Akashi Therapeutics announced the boy who developed serious, life threatening heath issues in their HALO trial has passed away.
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Top medical news stories this week
The Hereditary Neuropathy Foundation is partnering with Acetylon Pharmaceuticals to support preclinical research for Charcot-Marie-Tooth (CMT) syndrome.
Amarantus BioScience Holdings requested Rare Pediatric Disease Designation (RPDD) from the FDA for orphan drug MANF, for treating retinitis pigmentosa (RP).
Catabasis Pharmaceuticals and the Muscular Dystrophy Association (MDA) have collaborated to support Part B of the MoveDMD clinical trial of CAT-1004, a novel product candidate for the treatment of patients with Duchenne muscular dystrophy (DMD).
I think the entire Duchenne community has gotten a true glimpse of FDA communications, and we should all be deeply troubled by it.
Vertex Pharmaceuticals received a Complete Response Letter from the FDA for its sNDA for kalydeco in people with CF who have 1 of 23 residual function mutations
PPMD shared the communication they received from Eli Lilly stating the company has halted its studies examining the effect of tadalafil in Duchenne patients