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Work has begun in Professor George Dickson’s lab to refine the gene therapy product.Read more
Pharnext is carrying out an observational study for people with Charcot-Marie Tooth disease using a new digital app called CMT&Me.
Novartis has announced that the US Food and Drug Administration (FDA) has accepted its marketing application for AVXS-101 for the treatment of SMA type 1.
A recent study has demonstrated the value of a new mouse model of facioscapulohumeral muscular dystrophy (FSHD).
Stealth BioTherapeutics has initiated a phase 3 trial to test elamipretide in individuals with primary mitochondrial myopathy.
An international team of experts have published consensus-based care recommendations to help clinicians deliver high standards of care to adults with myotonic dystrophy 1.
The US based charity, Neuromuscular Disease Foundation, has been awarded a challenge grant to support gene therapy research for individuals with GNE myopathy.
AveXis has announced it is seeking marketing approval for its gene therapy, AVXS-101, in Europe, United States and Japan for infants with SMA type 1.
Work has begun in Professor George Dickson’s lab to refine the gene therapy product.
Sarepta Therapeutics has announced that it will be working with Dr Zarife Sahenk from Nationwide Children’s Hospital in the United States to develop a gene therapy for CMT.
PTC Therapeutics recently presented positive preliminary data on the long-term clinical benefit of Translarna at the World Muscle Society conference.