Rare Diseases and Orphan Drugs
Rare diseases affect a small percentage of the population but collectively impact millions worldwide. Orphan drugs, developed specifically for these conditions, face unique challenges due to the limited market size. However, incentives such as the Orphan Drug Act and global collaborations have significantly boosted research and development. Despite high development costs, orphan drugs represent a lifeline for patients with otherwise untreatable diseases. The pathway from discovery to market is complex, involving rigorous regulatory scrutiny, innovative trial designs, and patient advocacy support to ensure that these drugs are both safe and accessible.
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Rare Diseases and Orphan Drugs Conference Speakers
Recommended Sessions
- Advancement in the Diagnosis of Rare Diseases
- Biomarkers for Rare Disease Diagnosis and Treatment
- Biomedical Research
- Challenges of Living with a Rare Disease
- Clinical Case Reports and Case Studies
- Clinical Research on Orphan Drugs
- Different Types of Rare Diseases
- Drug Discovery for Rare Diseases
- Emerging Technologies in Rare Disease Research
- Functional Genomics and Gene Therapy for Rare Diseases
- Genetic Testing and Counselling of Rare Diseases
- Health Economics and Market Access for Orphan Drugs
- Infectious Diseases and Immune Deficiencies
- Introduction and Causes of New Rare Diseases
- Orphan Drugs Potentiality
- Rare Autoimmune Disorders
- Rare Bone Diseases
- Rare Cancer
- Rare Diseases and Orphan Drugs
- Rare Diseases of the Heart and Vessels
- Rare Neurological Disorders
- Rare Nutritional and Metabolic Diseases
- Rare Paediatric Diseases
- Rare Skin Diseases
- Regulatory Considerations for Developing Orphan Drugs
- Treatment and Advanced Therapies for Rare Diseases
- Vaccines and Vaccination for Rare Diseases
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