March 15, 2024 – On the occasion of International Rare Disease Day on February 28, the Swiss Foundation for People with Rare Diseases drew attention to the current challenges and needs of those affected. In Europe, a disease is considered …

Rare diseases: No diagnosis or the most expensive drug in the world? Read more »

December 20, 2017 –  The American Food & Drug Administration (FDA) has just approved  a novel gene therapy to treat patients with a rare form of inherited vision loss. Voretigene Neparvovec-Rzyl (Luxturna) is the first directly administered gene therapy approved to …

Voretigene Neparvovec-Rzyl (Luxturna): Gene therapy to treat inherited vision loss Read more »