The EveryLife Foundation for Rare Diseases commends the Food and Drug Administration (FDA) on today’s announcement of its intention to create a Rare Disease Innovation Hub. Co-chaired by CDER Director, Dr. Peter Marks, and CBER Director, Dr. Patrizia Cavazzoni,...
Michael Pirovolakis was diagnosed with spastic paraplegia type 50 (SPG50) when he was 18 months old. SPG50 is an “ultra-rare” progressive neurodegenerative disorder that causes developmental delays, speech impairment, seizures, a progressive paralysis of all four...
On June 6th, Moderna announced that its investigational therapy for methylmalonic acidemia (MMA), mRNA-3705, has been chosen for the FDA’s Support for Clinical Trials Advancing Rare Disease Therapeutics (START) pilot program. First launched in September 2023, the...
Ultragenyx moved one step closer to establishing a new surrogate biomarker for accelerated approval in rare disease gene therapies. The biotech said Wednesday it will seek accelerated approval for its Sanfilippo syndrome type A gene therapy after the FDA appeared...
The START pilot program aims to accelerate the development of novel drug and biological products for rare diseases rAAV-Olig001-ASPA was selected as one of a few CBER-regulated products based on eligibility criteria, including clinical benefit for rare diseases with...
