Related coverage linked through entity extraction aliases.
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.
