Genethon Pursues Different Strategies for Ensuring Patient Access to Gene Therapies for Rare Diseases
Genethon , a non-profit research organization focused on developing gene therapies for rare diseases, today marks International Rare Disease Day by highlighting its efforts to bring gene therapies to patients suffering from rare diseases such as limb girdle muscular dystrophies, Crigler Najjar syndrome and Duchenne muscular dystrophy.
- Genethon , a non-profit research organization focused on developing gene therapies for rare diseases, today marks International Rare Disease Day by highlighting its efforts to bring gene therapies to patients suffering from rare diseases such as limb girdle muscular dystrophies, Crigler Najjar syndrome and Duchenne muscular dystrophy.
- Globally more than 300 million people, most of them children, are living with 7,000 rare diseases.
- Because patient populations are small, these diseases don’t readily fit into the biopharma industry’s business models.
- “Despite challenges in generating interest with biopharma companies and investors, Genethon will never abandon patients suffering from rare and ultra-rare diseases,” said Dr. Revah.