DMD
Sarepta Therapeutics gains expanded FDA approval for DMD treatment
June 21, 2024Medical Communications DMD, Musculo-skeletal disorder, Sarepta Therapeutics
Sarepta Therapeutics has announced that the US Food and Drug Administration (FDA) has approved an expansion to the labelled indication …
Pfizer shares update on DMD gene therapy clinical trial
June 13, 2024Research and Development DMD, Musculo-skeletal disorder, Pfizer, duchenne muscular dystropy, gene therapy
Pfizer has announced an update from its phase 3 randomised, multicentre, double-blind, placebo-controlled CIFFREO trial, which assessed the investigational mini-dystrophin …
Santhera announces Early Access Program by partner Sperogenix for DMD treatment
June 11, 2024Medical Communications DMD, Musculo-skeletal disorder, early access, muscular dystrophy, santhera
Santhera Pharmaceuticals has announced that its partner Sperogenix Therapeutics has launched a paid-for Early Access Program (EAP) for Agamree (vamorolone) …
FDA turns down Sarepta’s DMD drug over safety concerns
August 20, 2019Manufacturing and Production DMD, FDA, approval safety, pharma, sarepta
The FDA has declined to approve Sarepta Therapeutics’ Duchenne muscular dystrophy (DMD) drug, citing safety concerns including risk of infection …
Breakthrough gene therapy shows promise in Duchenne muscular dystrophy
July 26, 2017Research and Development DMD, Duchenne Muscular Dystrophy, University of London, University of Nantes, biotech, drugs, pharma, pharmaceutical
New research, led by teams from AFM-Telethon laboratory in Nantes and the University of London, have developed a gene therapy …
US panel says Sarepta’s trial DMD drug not proven effective
April 26, 2016Manufacturing and Production, Research and Development DMD, US FDA, drug trial, sarepta
A US advisory committee has concluded Sarepta Therapeutics’ (Nasdaq: SRPT) trial drug to treat muscle wasting condition has not proved …
NICE recommends PTC Therapeutics’ Translarna to treat Duchenne muscular dystrophy
April 18, 2016Sales and Marketing DMD, Duchenne Muscular Dystrophy, NICE, PTC Therapeutics, Translarna, regulation
Shares in drug maker PTC Therapeutics (Nasdaq: PTCT) jumped after the National Institute for Health and Care Excellence (NICE) backed …
UK life sciences gains £93 million backing
July 31, 2013Research and Development, Sales and Marketing BIA, DMD, life sciences
The UK life sciences sector has been given a new £93.2 million package of support by the government to help …
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