Credit: Unsplash/CC0 Public Domain A collaborative effort between investigators at the National Institutes of Health’s National Institute of Allergy and Infectious Diseases (NIAID) and Massachusetts General Hospital (MGH) demonstrates the potential of precise...
Last week, Eli Lilly opened the doors of its new research center in Boston, largely focused on genetic therapies. While the Indianapolis-based drugmaker’s $875 billion valuation largely stems from its diabetes and weight loss medicines, CSO Dan Skovronsky said that...
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...
