Rare Diseases and Orphan Drugs

Bryan Gutierrez has completed his Bachelor degree in Pharmacy at the University of Santo Tomas. He is currently a pharmacist at Al Zahra Private Hospital Dubai. He specializes in the knowledge, handling, and extemporaneous compounding of chemotherapy medications and regimens. He designed the Pharmacology in Critical Care Course which include basic and clinical pharmacology in the critical care. Clinical Pharmacist-In-Charge, First Successful All-Filipino Pediatric Liver Transplant in the Philippines.

chemotherapy medications and regimens, Clinical pharmacology

Lecturer and medical oncology specialist in Clinical Oncology department, Faculty of medicine, Suez Canal University Hospital, Ismailia, Egypt. Author at Medical Fraternity – an online super-speciality physician case based learning platform. Ph.D. degree in Clinical and Experimental Oncology and Immunology, department of Surgery, Oncology and Gastroenterology, Padua University and Veneto Oncology Institute, Padua, Italy. I was involved in basic research, clinical activities and following clinical trials. Courses studied are the following: Oncology, Cancer virology, Oncohematology, Tumour immunology, Genetics and Bioinformatic analyses, Methods of imaging in medicine and biology, Biostatistics, Bioethics, Bibliometrics, Communicating science effectively.

Oncology, Cancer virology, Genetics, Methods of imaging in medicine and biology, Tumour immunology

Consultant thoracic and breast oncological surgery through the period 1991-2018 in Latakia national hospital, ministry of health. I was named the chief of residency training program in Latakia directorate of health in 2010. I was appointed chief of surgical departments, international member of American society of Breast surgeon, lecturer and founder member of the Syrian foundation for breast cancer support in Latakia. Private experience in breast cancer, screening, detection and surgery.

Thoracic, Breast oncological surgery

Jeff Leider, of Elmwood Park, New Jersey, has been on the forefront of Rare Disease Awareness for a little over five years now. His passion for advocacy began when his sons were diagnosed with MPS II/Hunters Syndrome. Since that heart wrenching day of diagnosis, he has worked tirelessly not only for his children but all children with Rare Diseases. Jeff is the President and Founder of the Let Them Be Little X2 Foundation. Mr. Leider has been on the forefront of bringing legislation to our governmental agencies. With Mr. Leider’s assistance and leadership a new law, The Let Them Be Little Act, providing testing of MPS I & MPS II in newborns, was recently signed into law by Governor Chris Christie. This is only the second state in the Union to implement this law. Mr. Leider has also been an integral part, with the support and guidance of Congressman Bill Pascrell (D-NJ9), in persuading the FDA and Shire Pharmaceuticals to open a clinical trial, allowing nine children diagnosed with Hunters Syndrome a chance to receive experimental drug to halt disease progression. Locally Mr. Leider is a Board Member for the Elmwood Park Youth Football Bombers and Officer of the Elmwood Park Little League. In November 2014, Mr. Leider was the award recipient of The Abby at the Rare Voice Awards in Washington, DC for advocacy at the State Level.

Rare Disease Awareness

Dr Shrutti Dhar currently working as Country Brand Lead for Rare Diseases India. In this role she is responsible for all the Pfizer rare diseases products and work actively to launch them in India and provide access to those patients who need it. Her areas of focus are Hemophilia, Sickle cell disorders and Gauchers Diseases for which Pfizer has an exciting pipeline of new products. Previously she was responsible for Medical affairs for Rare Diseases India. As a Medical Lead she was actively working with key decision makers in Government and private sector to Increase awareness, improve diagnosis and provide access to rare diseases therapies.

Hemophilia, Sickle cell disorders and Gaucher's Diseases

I am Dr.Mojtaba Mafi M.D., Medical Doctor/ Physician, Graduate of Tehran university, School of Medicine with honor. I passed post graduate courses on Nutrition, Gastroenterology and hepatology, Obesity treatment and diabetes approved by Medical sciences university successfully. I am associate of Iran Hepatitis Network and Gastroenterology and Hepatology research center, Middle East Liver Diseases Center (MELD). I studied on fatty liver and related metabolic disorders among Iranian families from 2015 and currently publishing the results of our study and research about fatty liver and hyperlipidemia, diabetes and nutrition as study director and clinician associate member of Hepatitis Network. I am reviewer of Hepatitis Monthly Journal and journal of Clinical and Translational Hepatology. I passed successfully more than 10 courses with certificates in Iranian association of gastroenterology and hepatology. One of my works on diabetes and fatty liver as a scientific article has been accepted in 4th International Conference on Diabetes and Its Treatments at Dubai 2018 for oral presentation and publication. I am organizing committee member and speaker of International Conference of Nutrition and Diet Imbalance, Indonesia, 2018. My article on fatty liver and metabolism has been accepted in International Conference on Gastroenterology and hepatology, Thailand 2018, for oral and poster presentation. I am verified reviewer and certified mentor of Publons academy in the field of Clinical Medicine and Hepatology. I am invited participant for International Conference of Translational Medicine, Hawaii, from editor in chief of American Journal of Translational Medicine. I am invited speaker of International Conference on Obesity and Chronic diseases, 2018 in Dubai.

Hepatology, Liver Diseases, Gastroenterology

She has Doctor Degree in Microbiology and Immunology. She has established the Quality Control Program of vaccines and the Brazilian Biosafety Program at the Ministry of Health in Brazil, where she has worked for 38 years. She has mentored quality control and biosafety trainings as an international consultant. She has founded the Brazilian Biosafety Association and the International Federation of Biosafety Associations. She was Chairperson of the Brazilian Biosafety Committee and has represented Brazil in several international agreements. She was adviser to many countries on Biosafety of Biotechnology procedures and she was awarded by the International Federation of Biosafety Association as Biosafety Hero in 2015.

Biotechnology

Dr. Neusa O.V.T Lopes, MD, Pulmonologist, is a graduate of Christian University Medical School, Jakarta Indonesia as a medical doctor. 2015 she finished her specialization in Respiratory medicine at the University of Indonesia. Dr. Neusa has been practicing since 2004, the last 10 years at National Hospital Guido in Dili, Timor-Leste, one of the Timor’s top referral hospitals, where in her practice, she specializes in the care of critically ill, respiratory diseases. She is board certified in pulmonary medicine and respiratory disease by Indonesia medical board. She also a President of the Multi Drug Resistant Tuberculosis Program of Ministry of Health, East Timor. Certified by the FDA for Clinical Trials and a member of the Conduct Clinical Trial program, based in California, USA. Dr. Neusa is a member of Indonesia pulmonologist association and Timor-Leste medical association. Her first research, entitled, “Cohort Review Six months of MDR TB in Timor-Leste and Persahabatan Hospital” was published in Journal of Respiratory in Indonesia. In addition, her second research study with the title “Lung Cancer in Young Males”, was published in the Respiratory Journal in Sydney, Australia, and in ECRON, London. Her research interests are related to lung cancer occurring at young ages, drug resistant tuberculosis and advance tuberculosis. She strongly believes that understanding the basic physiology, pathophysiology, immunology and genetic inherent also genetic mutation of the disease will ultimately lead to better holistic management or even a cure for cancer and drugs resistant.

Lung cancer,Drug-resistant tuberculosis, Immunology, Pathophysiology

Dr. Ali is a Clinical and Molecular geneticist certified by the American Board of Medical Genetics and Genomics (ABMGG), currently serving as medical director in global clinical development at Ultragenyx pharmaceutical in California, USA. Dr. Ali’s experience includes clinical genetics practice [full-time/USA previously and telemedicine/MENA currently], leadership and directorship roles at a CLIA-CAP certified DNA diagnostic laboratory/USA [previously], and translational research experience at the National Institutes of Health (NIH)/USA [previously]. Dr. Ali also sits on the gene editing task force/gene therapy committee at Alliance for Regenerative Medicine (ARM) and pediatric expert work group at the International Conference on Harmonisation (ICH).

Gene therapy, Pediatric expert

Samer Ellahham has served as Chief Quality Officer for SKMC since 2009. In his role, Dr. Ellahham has led the development of a quality and safety program that has been highly successful and visible and has been recognized internationally by a number of awards. As Chief Quality Officer and Global Healthcare Leader, he has a focus on ensuring that that implementation of this best practices leads to breakthrough improvements in clinical quality and patient safety. He Ellahham is a recognized leader in quality, safety, and the use of robust performance improvement in improving healthcare delivery. He serves on a number of US and international committees and advisory bodies. Samer Ellahham is Certified Professional in Healthcare Quality (CPHQ) by The National Association for Healthcare Quality (NAHQ). He is certified in Medical Quality (CMQ) by The American Board of Medical Quality (ABMQ). He is the recipient of the Quality Leadership Award from the World Quality Congress and Awards and the Business Leadership Excellence Award from World Leadership Congress in 2015. He is one the nominees for Safe Care magazine Person of the Year in the United States

heart failure, genetics, cardiac imaging, sports and nutrition, innovation, safety, outcomes research, risk management, safe surgery.

Dr. Mohamad Miqdady is American Board certified in Pediatric Gastroenterology, Hepatology and Nutrition. He is the Division Chief, Ped. GI, Hepatology & Nutrition Division at Sheikh Khalifa Medical City in UAE. Program Director, Pediatric Gastroenterology Fellowship Training program, SKMC, Abu Dhabi, UAE. Also an Adjunct Staff at Cleveland Clinic, Ohio USA. Expert member of the FISPGHAN Council (Federation of International Societies of Pediatric Gastroenterology, Hepatology, and Nutrition); Malnutrition/Obesity Expert team. Dr. Miqdady completed his Fellowship in Pediatric Gastroenterology at Baylor College of Medicine and Texas Children’s Hospital in Houston, TX, USA. He held the position of Assistant Professor at Jordan University of Science and Technology in Jordan for six years prior joining SKMC. Main research interests include nutritional disorders, feeding difficulties, picky eating, obesity, procedural sedation, allergic GI disorders and celiac disease. He has 20 publications in peer reviewed journals. On the Editorial Board of few journals including Gastroenterology & Hepatology.

Pediatric Gastroenterology, Hepatology and Nutrition

Yavuz is the chairman of Istanbul Consulting Group. ICG(Istanbul Consulting Group) was founded in 2013 and provided guidance to the Turkish ministry of health as part of a World Bank project.Yavuz is currently the Co-Founder of BioCube İstanbul Bioentrepreneurship & Innovation Center and Corporate Communication Director of Sumitomo Group/ Expel İlaç Archem Diagnostices Endmed and Diagen. He previously managed the largest distributor of Siemens Healthcare in Turkey managing 250 employees and director of Avcılar Hospital R&D Center, Chief Medical Officer of Lifematrix GmbH. Previously he worked as the Market Access & Health Policy Director for AIFD in Turkey. Yavuz previously worked as the Vice President of Ipsen pharmaceutical and Director of Teva pharmaceutical in USA managing large clinical trials as well as Investigator Initiated Trials and developing relationships with Key Opinion Leaders. Previously, Yavuz was the Associate Director at KV Pharmaceuticals and Director in Clinical Development department at Forest Laboratories. Yavuz earned his MD from the Faculty of Medicine, University of Ankara in Ankara, Turkey. He completed a clinical internship at Baylor College of Medicine in Houston, followed by continued research training at The University of Texas MD Anderson Cancer Center in Houston. He recently completed his Executive MBA at the Olin Business School at Washington University in St. Louis. Yavuz currently resides with his wife Dr. Kamile Sılay and their two daughters and one son in Ankara,Turkey.

Pharmaceutical, Biotech

Dr Timothy Low is currently the Head of Medical Affairs Asia Pacific at Shire, a position he assumed on October 2, 2017, based in its regional headquarters in Singapore. He was awarded 2018 Rare Disease Champion Provider of the Year in Asia Pacific. Concurrently in 2018, he was also conferred the Icons of Healthcare – Leading Healthcare Management Professional of the year. Prior to joining Shire, Dr Low served for two years as Chief Executive Officer (CEO) of Farrer Park Hospital, a private acute tertiary institution sited within Singapore’s first fully integrated healthcare and hospitality complex. It was awarded “Best New Hospital in Asia Pacific” and “Smart Hospital of the Year in Asia Pacific” in 2016/2017 respectively. He currently serves as a member of the hospital’s Board of Directors. With 30 years tenure in both public and private practice, Dr Low brings to his role his operational and medical knowledge, along with his extensive strategic experience leading teams and bringing specialty care to patients. He formerly held the positions of CEO of Gleneagles Hospital from 2000 to 2006 and as Medical Affairs senior executive across pharmaceutical and medical device industries at Covidien, Schering Plough, Covance and GlaxoWellcome, spanning more than 17 years. Dr Low joins the prestigious ranks of Stanford Who’s Who for his contributions toward the healthcare community. He was also awarded the “50 Most Talented Healthcare Leaders of Asia” award as well as “Best CEO of the Year” at the Asia Healthcare Excellence Award conferred by CMO Asia in 2016 and 2017 respectively. Dr Low received his Bachelor of Medicine and Bachelor of Surgery from the National University of Singapore (NUS) and started his professional career in Public Service at various local hospitals and as a General Practitioner at Raffles Medical Center. He also holds various graduate degrees awarded by the NUS Graduate School of Business, Stanford University Executive Programme and the Singapore Management University Asia Pacific Hospital Management Programme.

Rare Disease

Dr Stef Stienstra works internationally for several medical and biotech companies as scientific advisory board member and is also an active reserve-officer of the Royal Dutch Navy in his rank as Commander (OF4). For the Dutch Armed Forces he is CBRNe specialist with focus on (micro)biological and chemical threats and medical- and environmental functional specialist within the 1st CMI (Civil Military Interaction) Battalion of the Dutch Armed Forces. For Expertise France he is now managing an EU CBRN CoE public health project in West Africa. He is visiting professor for the University of Rome Tor Vergata in Italy for the CBRN Masters Course and lecturer for the NATO School in Oberammergau in Germany and the Joint NATO CBRN-Defense Center of Excellence in Vyskov in the Czech Republic. In his civilian position he is at this moment developing with MT-Derm in Berlin (Germany) a novel interdermal vaccination technology as well as a new therapy for cutaneous leishmaniasis for which he has won a Canadian ‘Grand Challenge’ grant. With Hemanua in Dublin (Ireland) he has developed an innovative blood separation unit, which is also suitable to produce convalescent plasma for Ebola Virus Disease therapy. He has finished both his studies in Medicine and in Biochemistry in The Netherlands with a doctorate and has extensive practical experience in cell biology, immuno-haematology, infectous diseaases, biodefense and transfusion medicine. His natural business acumen and negotiation competence helps to initiate new successful businesses, often generated from unexpected combinations of technologies.

cell biology, immuno-haematology, infectous diseaases, biodefense and transfusion medicine

Dr Samrat Shah has completed his MD(Internal Medicine) from KLE University-Karnataka. He is gold medalist in Internal Medicine. He has done Fellowship in Diabetology from Dr.Mohans Acadamy (IDF approved). He is presently working as Consultant Physician At Mediheal Group Of Hospitals. He has 6 International publications and several National and Regional publications.

Internal Medicine, Diabetology

Masoabi currently serves as a administrative coordinator for the South African Haemophilia Foundation and also serves as a Chairperson for the South African Haemophilia Foundation Youth Council.

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Dr. Abdul Matin received his PhD from Birkbeck, University of London followed by a Postdoctoral Fellowship from School of Medicine, Southampton University Hospital, Southampton, United Kingdom. His long-standing research interests include the epidemiology and pathogenic mechanism of emerging parasitic diseases with special interest on role of Blood-Brain Barrier in Central Nervous System infections. Using multi-disciplinary approach he is looking for potential novel synthesized compounds or nanoparticles or/and obtained from plants or insects to discover potential drug candidates or/and for drug delivery system to alleviate the burden of life threatening infections. He is conducting research using state-of-the-art technologies in the field of infectious diseases. He is recipient of various international and national awards, honors and grants. He is currently Associate Professor at Majmaah University, Saudi Arabia. He has published >36 research articles in reputed journals and is serving as an editorial board member and reviewer for various international and national reputed journals and funding agencies.

parasitic diseases

Dr. Ellahham has served as Chief Quality Officer for SKMC since 2009. In his role, Dr. Ellahham has led the development of a quality and safety program that has been highly successful and visible and has been recognized internationally by a number of awards. As Chief Quality Officer and Global Healthcare Leader, Dr. Ellahham had a focus on ensuring that that implementation of these best practices leads to breakthrough improvements in clinical quality, patient safety, patient experience and risk management. Dr. Ellahham is a Board-certified internist, cardiologist and vascular medicine senior consultant and continues to care for patients. He received his undergraduate degree in biology and his M.D. from the American University of Beirut, Beirut, Lebanon.

Cardiology

She currently holds the positions of Senior Executive Vice President of MaxifyLife, Corporate Engagement Manager of the Jansen’s Foundation, Global Gene’s Foundation Alliance Council Member and Founding Board Member of Rare is Not Rare. Recently, Nadia has also facilitated and coordinated the development of the Rare Advocacy Movement (RAM), a patient advocacy initiative focused on documenting the ever-evolving complex structure and unique dynamics of the rare disease patient advocacy landscape.

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Strategic and creative consultant in biomedical science, with a parallel career in the Dutch Civil-Military Interaction Command in which he has responsibility for the counter measures in CBNRe threats and (medical) consequence management both in a military and a civilian (terrorism) setting. He was the director of the 2014 & 2016 World Congress of CBRNe Science & Consequence Management in Tbilisi, Georgia. He works internationally as consultant or scientific supervisory board member for several medical and biotech companies, merely involved in biodefense, clinical diagnostics and therapies. He is also visiting professor for Punjab University in Pakistan and Rhein-Waal University in Germany and visiting professor at the University of Rome Tor Vergata. He has finished both his studies in Medicine and in Biochemistry at the University of Groningen in The Netherlands and has extensive practical experience in cell biology, immuno-haematology, biodefense and transfusion medicine. His natural business acumen and negotiation competence helps to initiate new successful businesses, often created out of unexpected combinations of technologies. His thorough understanding of abstract science combined with excellent skills in the communication of scientific matters to non-specialists, helps him with strategic consulting at top level management.

Ebola

Mr. Schliebner is a clinical strategist with a 25-year background in clinical drug development focused on rare diseases, orphan drugs, and advanced therapeutics. He develops innovative solutions and strategic considerations to help overcome the operational, logistical, and ethical challenges inherent to conducting research with rare disease patients. Mr. Schliebner’s efforts are focused on patient-centered approaches to drug development; reducing the burden of clinical trial participation; and leveraging novel and disruptive technologies to bring new therapies to patients faster. Committed to collaborative efforts to further drug development, Mr. Schliebner has developed active partnerships with rare disease patient-focused organizations, patient advocacy groups, drug development institutes, and academic research centers. He serves on leadership committees for a variety of rare disease societies and congresses, and also regularly speaks on novel strategies to accelerate rare disease drug development. Previously, Mr. Schliebner served as the Chief Operating Officer for an international cancer research institute focused on developing tumor-specific research consortia to accelerate the development of new targeted therapies. He holds a Master’s Degree in Public Health (MPH) from the University of Utah School of Medicine, and completed a Graduate Research Fellowship at The National Institutes of Health.

rare diseases, orphan drugs, and advanced therapeutics

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I earned a Medical Doctorate (MD) in Community Medicine, Master Degree in Public & Tropical Health, Master Degree in Tropical Medicine & Infectious Diseases, Postgraduate Diploma in Public Health, Certificate of Diploma Course in Epidemiology & Biostatistics and a Bachelor’s degree in of Medicine & Surgery. Currently, I am working in the UAE as a Community Medicine Specialist, at Central Directorate of Preventive Medicine with Ministry of Health & Prevention where under the National Program of Expanded Immunization (EPI), I am managing National Poliomyelitis Eradication Program and National Program for Elimination of Measles and Rubella by following guidelines of Global Polio Eradication Initiative (GPEI) and the World Health Organization (WHO). Also I am technically supporting the National EPI program in maintaining high immunization coverage against: Poliomyelitis, Measles and Rubella. Conduction of random supervision visits is a key responsibility to me as implemented active surveillance activities at high priority sites could be verified.

Epidemiology

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Pediatric, Endocrinology

Alba Ancochea is the CEO of Spanish Federation of Rare Diseases and its Foundation. She is a EURORDIS board of Directors member , as well as member of the Rare Diseases International Advocacy Working Group, member of the DITA (Drug Information ,Transparency and Access) Task Force, representative of the Council of National Alliances and supporting the Ibero-American Alliance of RD. She belongs to the CIBERER (Spanish Biomedical Research Consortium on RD )Scientific Advisory Board. She has completed undergraduate and masters studies in psychology, special needs teaching and management of NGOs.

Drug Development and Clinical Research

Alastair Kent was the executive director of Genetic Alliance UK, the British umbrella body for over 200 patient groups supporting that with rare and genetic disorders. Since retiring from Genetic Alliance UK he has continued his involvement as an independent advocate in health policy and service development that will improve support for those affected or at risk from these conditions.

rare and genetic disorders

Don Van Dyke is Chief Operating Officer at Cloud Pharmaceuticals. In his earl career, he held increasingly responsible general management, business development, and executive positions at American Hospital Supply, Dade Diagnostics, Eastman Kodak and Millipore Corporation.Van Dyke's work at the intersection of biological science and computer science began at startup Bio Image Corporation. The company became the market leader in image-based genomics and proteomics under his leadership as VP sales, VP worldwide marketing and sales, general manager, and CEO. The company merged with another firm and went public on the NASDAQ. He has had executive roles at Molecular Mining Corporation, a life science data mining company and INCOGEN, a Bioinformatics company and has advised more than a dozen start-ups and government agencies.

Sara Tylosky, CEO of Farmacon (www.farmacon.co) has over twenty years of experience in strategic management & global marketing in the pharmaceutical & biotech industries. Farmacon works with sponsors, from large pharmas to start up biotechs, through their network of expert consultants to tailor solutions for clinical trial research projects. Working in phase 2-4 trials, their model is to dispatch expert physicians and logistical support on the ground in each country to bring real-time feedback, provide strategic intelligence, uncover issues, and resolve problems to reach enrolment early and reduce study costs. This allows for more accurate data to be collected. Their particular specialty is in rare disease for projects that need assistance outside of us. Sara holds a master’s in business from Florida Atlantic University and an undergraduate degree from Whitman College.

Michael S. Wolfe received his B.S. in chemistry from the Philadelphia College of Pharmacy and Science and Ph.D. in medicinal chemistry from the University of Kansas. After postdoctoral stints at the University of Kansas and the NIH, he joined the faculty of the University of Tennessee in Memphis. In 1999, he moved to Harvard Medical School and Brigham and Women’s Hospital, where his work focused on understanding the molecular basis of Alzheimer’s and related disorders and identifying effective approaches for pharmacological intervention, becoming Professor of Neurology in 2008. He returned to the University of Kansas in October of 2016 as the Mathias P. Mertes Professor of Medicinal Chemistry. Awards for his work include the Sato Memorial International Award in bioorganic and medicinal chemistry from the Pharmaceutical Society of Japan, the MetLife Award for Biomedical Research, a Zenith Fellows Award from the Alzheimer’s Association, and the Potamkin Prize from the American Academy of Neurology.

Alzheimer’s Disease

Dr. KrupaTorne is among the few super-specialists who have been internationally trained in the field of Paediatric Neurology. At present, she is heading the Department of Neurosciences at Surya Children’s multispecialty Hospital in Mumbai, India. She has completed her Fellowship in Paediatric Neurology and Epilepsy from Maharashtra University Health Sciences at Lokmanya Tilak Municipal Hospital and Lokmanya Tilak Municipal Medical College, Mumbai. She has had a brief stint at the University College London Hospital, a premier academic institution cum hospital in the UK. She has been trained in Paediatric Neurology at Birmingham Children's Hospital where she successfully completed her International Fellowship in Paediatric Neurology, Epilepsy and Neurorehabilitation. She was honored by International Bursary from European Paediatric Neurology Society with an opportunity to attend one of the prestigious courses in Paediatric Neuromuscular disorder and Neonatal Neurology at Budapest in Hungary. She is affiliated with many International and National Paediatric Neurology and Epilepsy associations.

Her research interest is in complex paediatric epilepsies, ketogenic diet and paediatric movement disorder.

Tiziana Greggi Specialized works in Orthopedics and Traumatology, at the University of Bologna, Specialized in Biomedical Technologies, at the University of Bologna and she is the Head of Simple Departmental Structure: Spinal deformity surgery. She has worked in the Division of Spine Surgery of the Rizzoli Institute, first as an assistant and then as a senior manager. From 2000 to 2008 he received temporary positions as Director of a complex structure. From the end of this position to date has been responsible for the Organizational Module "the spinal deformities in pediatric age and associated with rare diseases" in the same Division. He is also responsible for a specialized clinic dedicated to the study and treatment of severe spinal deformities associated with rare diseases at the Rizzoli Orthopedic Institute. He is currently Head of the Departmental Simple Structure: Spinal deformity surgery

Spinal deformit, Spinal Surgery

Dr. Bhat has over 18 years’ experience in drug discovery and development and prior to founding Reviva, he held research positions at XenoPort, ARYx Therapeutics and Higuchi Biosciences Center in the United States. Dr. Bhat has received a global post-doctoral training at the University of Kansas, USA, the Georg-August-Universität, Göttingen, Germany and the Université du Maine, France. In 1995, he was selected for the Alexander von Humboldt fellowship, an internationally recognized award for young scientists to pursue advanced research in Germany. Dr. Bhat received his Ph.D. in synthetic organic chemistry from the Central University (NEHU), India and has authored over 15 research papers published in peer-reviewed scientific journals. Dr. Bhat is an inventor on more than 100 filed patents and over 35 granted patents

Drug Discovery

The Department of Department of Infectious Diseases / Tropical Medicine, Nephrology and Rheumatology at St. Georg Hospital in Leipzig is about regional focus clinic and competence center for infectious diseases for the state of Saxony

Infectious Diseases & Resarch

Stef Stienstra works internationally for several medical and biotech companies as scientific advisory board member and is also an active reserve-officer of the Royal Dutch Navy in his rank as Commander (OF4). For the Dutch Armed Forces he is CBRNe specialist with focus on (micro)biological and chemical threats and medical- and environmental functional specialist within the 1st CMI (Civil Military Interaction) Battalion of the Dutch Armed Forces. For Expertise France he is now managing an EU CBRN CoE public health project in West Africa. In his civilian position he is at this moment developing with MT-Derm in Berlin (Germany) a novel interdermal vaccination technology as well as a new therapy for cutaneous leishmaniasis for which he has won a Canadian ‘Grand Challenge’ grant. With Hemanua in Dublin (Ireland) he has developed an innovative blood separation unit, which is also suitable to produce convalescent plasma for Ebola Virus Disese therapy. He has finished both his studies in Medicine and in Biochemistry in The Netherlands with a doctorate and has extensive practical experience in cell biology, immuno-haematology, infectous diseaases, biodefense and transfusion medicine. His natural business acumen and negotiation competence helps to initiate new successful businesses, often generated from unexpected combinations of technologies.

Infectious Diseases

An expert in international health initiatives, health in developing nations, HIV/AIDS research

Infection Diseases, Neglected Diseases

Nadia Ameen completed her medical degree from the University of the West Indies, Jamaica. She completed training in Pediatrics at Children’s Hospital Wisconsin and then pursued post-doctoral fellowship in gastroenterology at Yale University School of Medicine. She is the principal investigator of an NIH supported research laboratory at Yale University that studies CFTR and diarrheal diseases for over 20 years. She was the first to recognize a trafficking defect in MVID before a genetic defect was identified. She has authored more than 30 papers in reputed journals and serves as an editorial board member for several journals.

Intestinal Diseases

Deputy Chairman, Intensive Care Department, Consultant, Pulmonary and Critical Care Medicine, Professor, King Saud Bin Abdulaziz University for Health Sciences, Riyadh, Kingdom of Saudi Arabia, Dr.AlDawood graduate from College of Medicine in Kingdom of Saudi Arabia. Then he completed the Residency in Internal Medicine Program in McMaster University in Canada He obtained Critical Care and Respirology Fellowship Program in McMaster University, Canada (Jul 97-Jun 00). In July 2000, he joined King Abdulaziz Medical City, Riyadh, Saudi Arabia as a Consultant in Critical Care and Pulmonary Medicine up to the present. He is currently the Deputy Chairman of the Intensive Care Department and Professor in King Saud Bin Abdulaziz University for Health Sciences. In addition, he has more than 40 publications including articles in the New England Journal of Medicine (NEJM), JAMA, American Journal of Respiratory Critical Care Medicine, BMC Anesthesiology, and Critical Care Medicine.

Respiratory Critical Care Medicine, Rare Diseases, BMC Anesthesiology,and Critical Care Medicine

Dr. Wei Zheng received his Ph.D. in Pharmacology from the State University of New York at Buffalo. He had worked 12 years in pharmaceutical companies (Berlex, Amgen, and Merck) for drug discovery and development. Since 2005, Dr. Zheng has worked at National Institutes of Health for development of therapeutics for rare and neglected diseases. He has identified several drugs from drug repurposing screens and advanced two of them to clinical trials. Currently, Dr. Zheng is working on the therapeutics development for lysosomal storage diseases, drug resistant cancer and infectious diseases.

Drug resistant, Infectious Diseases

Division of Bioinformatics and Biostatistics, FDA

Rare Diseases,chemoinformatics, Predictive toxicologyOrphan Drugs,

Professor Joseph Ndung’u is the Head of FIND’s Neglected Tropical Diseases (NTD) programme. He has a degree in veterinary medicine and surgery (BVM) from the University of Nairobi, and a PhD on the immunopathology of human African trypanosomiasis (HAT or sleeping sickness) from the University of Glasgow’s Veterinary School in Scotland. He became a Visiting Professor of the University in 2001, and has held a similar position at the University of Wisconsin, Madison, USA. In 2013, he became a Corresponding Fellow of the Royal Society of Edinburgh

Tropical Diseases

Dr. Yong Moon Choi completed his PhD from the State University of New York and his postdoctoral studies from Purdue University under 1979 Nobel Laureate Herbert C. Brown. He is currently the president and CEO of Bio-Pharm Solutions, based in South Korea. He founded SK Biopharmaceuticals (New Jersey, Seoul/DIT, Shanghai) in 1993, and while there, Dr. Choi discovered and developed drug candidates for central nervous system (CNS) disorders. Under Dr. Choi's leadership, SK Biopharmaceuticals was the first Korean company to receive an IND by the US FDA (YKP10A), and in total, Dr. Choi has received 10 IND's from the US FDA.

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Daniel Cohen , MD Phd, former Professor of Medical Genetics in Paris University has authored more than 150 peer reviewed papers in hire wired Journal , including the first integrated map of the Human Genome back in the 90’s. While he has discovered or co discovered numerous genes for rare and common diseases, he has also pioneered several key technologies like Large Scale Biology at Genethon, he cofounded in France, but also Pharmacogenomics, Genome Wide Association Studies and Network Pharmacology. He was a confounder of Millenium pharmaceuticals in Boston, MA. He is now Chairman, CEO and cofounder of the France Based company, Pharnext, focusing on treatment of unmet Neurodegenerative disorders by using Network Pharmacology.

orphan genetic

Serge Braun, PharmD, is Scientific Director of AFM-Telethon since 2005, the French Muscular dystrophy Association, and President of Genosafe, a CRO company dedicate to QC of biotherapeutic products. He obtained his degree in Pharmacy and a Ph.D in Pharmacology from the Louis Pasteur University of Strasbourg, France; was a postdoctoral fellow at the Neuromuscular Center, School of Medicine of the University of Southern California in Los Angeles, USA.

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In 2015 Dr. Arneric joined the Critical Path Institute as Executive Director of the Coalition Against Major Diseases (CAMD), a consortium focused on developing Drug Development Tools for advancing innovative treatments of Alzheimer’s disease and related dementias. Previously he was VP Research/Preclinical Development (Neuromed Pharmaceuticals), CSO of the Pain/Migraine Drug Hunting Team (Lilly), and held senior management positions at Pfizer, Pharmacia, DuPont Pharmaceuticals, and Abbott. He has extensive leadership and scientific expertise in the areas of neurology, pain, psychiatry and urology, and over the last 25 years his teams have delivered more than 30+ drug candidates into clinical development.

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Dr Stephen Shrewsbury is currently Executive Vice President of Development and Chief Medical Officer at Fortuna Fix, leading their novel cellular technology into multiple clinical programs focused on Regenerative Medicine, several of which are rare diseases. A UK trained physician, Dr Shrewsbury first joined Glaxo 23 years ago. He moved to lead inhaled antibiotic programs at Chiron Corporation (in Cystic Fibrosis and other rare diseases) before becoming CMO sequentially to MAP Pharmaceuticals, Adamas Pharmaceuticals, AVI BioPharma (now Sarepta Therapeutics – where he opened their first IND for eteplirsen in Duchenne Muscular Dystrophy and planned their oligomer programs against Ebola and Marburg Hemorrhagic Fevers) and Aquinox Pharmaceuticals. He has now moved from Drugs (and Devices) and Oligonucleotides to lead programs with directly reprogrammed precursor cells and tackle many rare, and currently untreatable, diseases with great social and healthcare impact. He is author of Defy Your DNA and over 70 scientific papers, holds several patents and contributed to many awarded grants – mostly for work on rare diseases.

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Yasutoshi Koga has completed his PhD at the age of 28 years from Kurume University and postdoctoral studies from College of Physicians and Surgeons of Columbia University (Profs. DiMauro S and Schon EA). He is the president of Japanese Society of Mitochondrial Research and Medicine, a Professor of Department of Pediatrics and Child Health, Kurume University School of Medicine. He discovered a novel therapeutic procedure and has completed the investigator-mediated clinical trial of L-arginine on MELAS in Japan. He received the Kelsey Wright Award from UMDF in 2008. In 2012, he started the new national project to cure the lactic acidosis associated with mitochondrial disorders, supported by Grants-in-Aid of the Research on Intractable Diseases (Mitochondrial Disorders) from AMED of Japan. He has published more than 120 papers in reputed journals and has been serving as an editorial board member of international journals.

DNA Sequencing, Clinical Research, Inherited metabolic disorders, New Drug Discovery, Clinical Genetics, Mitochondrial DNA

Gail Adinamis is founder and CEO of GlobalCare Clinical Trials, LLC, a global niche patient-centric service organization that takes study visits to the patient’s home or alternate setting via a global network of ambulant healthcare providers. Adinamis has over 35 years of comprehensive global clinical trials experience including over 12 years of global trials management at Abbott Laboratories and Astellas. She founded the in-home business model for study visits in 1992 and established and headed clinical trials divisions for three national home infusion companies and was founder, President and CEO of the first independent ambulant care service company for clinical trials. Adinamis is a member of the Women Presidents’ Organization, National Association of Professional Women, and DIA and has been an invited speaker at several industry conferences and recipient of numerous awards/recognitions including twice being among INC 5000’s fastest growing private companies, and Game Changer and CEO of the Year.

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Dr. Sahota works at Rutgers University in the following capacities: (i) professor in the Department of Genetics, (ii) director of scientific programs and director of the Clinical Genomics Laboratory, RUCDR Infinite Biologics; (iii) clinical professor and laboratory director in the Department of Pathology and Laboratory Medicine, Robert Wood Johnson Medical School (RWJMS); and (iv) clinical professor in the Division of Urology, RWJMS. Dr. Sahota’s research is focused on the molecular pathology of inherited urinary tract stone disease. He and his colleagues have characterized the molecular bases of adenine phosphoribosyltransferse (APRT) deficiency in the vast majority of Caucasian patients with this disorder.

Dr. Sahota’s research is focused on the molecular pathology of inherited urinary tract stone disease.

Pat Furlong is the Founding President and CEO of Parent Project Muscular Dystrophy (PPMD), the largest nonprofit organization in the United States solely focused on Duchenne muscular dystrophy (Duchenne). Their mission is to end Duchenne. They accelerate research, raise their voices in Washington, demand optimal care for all young men, and educate the global community.

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Prof Annemieke Aartsma-Rus obtained her PhD aged 27 at Leiden University (the Netherlands) in 2005. She became group leader in 2007 and is currently a professor of Translational Genetics at Leiden University Medical Center, the Netherlands. She currently chairs the TREAT-NMD Alliance (an infrastructure network for clinical trial readiness for neuromuscular disorders) and a networking Action (funded by Cooperation of Science and Technology (COST). She has published more than 100 papers in peer reviewed journals, has written multiple book chapters and has generated and maintains pages to explain Duchenne therapies in lay terms to the patient community. In 2009 she received the Duchenne award from the Dutch Duchenne Parent Project for her dedication to the field.

Duchenne muscular dystrophy