This award honors an emerging leader shaping the future of neuromuscular research will be presented at the 2026 MDA Clinical ...
Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS® ...
-- Company on track to report ELEVATE-44-201 Cohort 1 data in Q2 2026, with Cohort 2 data by end of year -- ...
Tokyo, Feb. 13 (Jiji Press)--An advisory panel to Japan's health minister decided Friday to allow public health insurance coverage for a gene therapy for muscular dystrophy beginning Feb. 20 and set ...
NMD Pharma A/S, a clinical-stage biotechnology company dedicated to developing novel therapies to restore skeletal muscle health, today announced that top-line data results from its Phase 2a ...
In a statement, PTC's chief executive, Matthew Klein, said the company had decided to pull the filing for Translarna (ataluren) as a treatment for nonsense mutation DMD after the FDA said it was ...
My twin sister spent years moving through consultations without a clear explanation for her symptoms. At one point, aged 31, ...
With the writing apparently on the wall, PTC Therapeutics has called off its latest bid for FDA approval of its Duchenne muscular dystrophy (DMD) drug Translarna. | Late Thursday, PTC revealed that ...
PTC Therapeutics withdrew its New Drug Application (NDA) resubmission for Translarna (ataluren) in nonsense mutation Duchenne muscular dystrophy (nmDMD) following feedback from FDA indicating that the ...
BASECAMP, a three-month, randomized, double-blind, placebo-controlled study, will evaluate SAT-3247’s safety and tolerability, and effect on muscle force, muscle quality and muscle regenerationTORONTO ...
Precision Biosciences Inc. has received FDA clearance of its IND application enabling clinical trial site activation for a phase I/II trial (FUNCTION-DMD) of PBGENE-DMD for the treatment of ambulatory ...
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