Treatment and Advanced Therapies for Rare Diseases

Advanced therapies, including gene and enzyme replacement therapies, provide hope for patients with rare diseases previously lacking treatment options. CRISPR and other gene-editing techniques aim to correct genetic defects at their source. RNA-based treatments and precision medicine approaches tailor interventions to individual genetic profiles. Continuous research, clinical trials, and international cooperation are essential to bring innovative treatments from the laboratory to the clinic. The integration of patient registries supports long-term tracking and improvement of therapeutic strategies.

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