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Jun 29, 2026
All 12 Rett syndrome patients treated with Taysha's TSHA-102 gene therapy in the REVEAL Phase 1/2 trial achieved at least one developmental milestone within one year, with functional gains continuing up to 30 months. The company has completed dosing in the Phase 3 portion and expects top-line data and FDA feedback in early 2027.
Jun 27, 2026
ACADIA Pharmaceuticals' stock jumped after the European Medicines Agency recommended approval of its Rett syndrome drug Daybue. The company also reported strong quarterly results and progress on its Alzheimer's disease psychosis treatment.
Mar 07, 2026
The FDA has denied or discouraged at least eight drug applications in the past year, including treatments for rare diseases, raising concerns about regulatory consistency and the future of experimental therapies.
Mar 05, 2026
The FDA granted Breakthrough Therapy designation to NGN-401, Neurogene's gene therapy for Rett syndrome, based on interim data showing durable functional improvements. The company expects to complete pivotal trial dosing in Q2 2026.
Mar 01, 2026
Artificial intelligence is revolutionizing rare disease diagnosis, cutting diagnostic timelines from years to weeks. Rare Disease Day on February 28 highlights challenges faced by over 300 million people worldwide living with more than 7,000 distinct rare conditions.
