Rare disease drug development

The author shares their experience of being diagnosed with limb girdle muscular dystrophy (LGMD) and highlights the lack of available treatments or cures. They emphasize the urgency for faster drug development due to the irreversible muscle loss caused by delays. The article notes advancements in gene therapies and FDA opportunities to modernize rare disease treatments.

Immunovant shares surge following positive arthritis trial data. UCSF researchers have submitted an FDA application for a prenatal gene therapy targeting a severe neurodegenerative disorder affecting young children. FDA leadership instability is causing investor concern and impacting rare disease drug development.