A research study using data from the UK Facioscapulohumeral muscular dystrophy (FSHD) Patient Registry has been published recently.Read more
The US Food and Drug Administration (FDA) has rejected PTC Therapeutics’ marketing application for Duchenne drug ataluren (Translarna).
Muscular Dystrophy UK is delighted to announce a one day training event for the volunteers and charity ambassadors who make such an enormous difference by supporting and represen
Scientists at the University of California, Berkley, have developed a new way to deliver the genome-editing technology, CRISPR-Cas9 into cells.
The US based company, Audentes Therapeutics, is currently developing a potential gene therapy (named AT132) for the treatment of X-linked myotubular myopathy (XLMTM).
A new version of CRISPR/Cas9 has been used to remove the toxic RNA that causes myotonic dystrophy.
The US-based company, AveXis, will be initiating a multi-centre trial named STRIVE. This study will be testing their gene therapy, AVXS-101, in babies with SMA type 1.
Acceleron Pharma has released an update on its neuromuscular programme. It is currently developing two drugs that aim to increase muscle mass by blocking myostatin.
Two research studies using data from the UK Myotonic Dystrophy Patient Registry have been published recently. The registry is jointly supported by Muscular Dystrophy UK and MDSG.
"This is disappointing but not unprecedented in the regulation process. Another Duchenne drug, Translarna, was initially rejected at an early stage but is now available."
Results from an MDA-supported study show that progressive resistance exercise is safe and can significantly reduce muscle weakness in children with CMT.