Researchers co-funded by Muscular Dystrophy UK and The SMA Trust have discovered a new role for the SMN protein, which is reduced in people with spinal muscular atrophy (SMA).
We’re excited to announce a new funding partnership with US-based charity, Cure CMD, to fund research into LMNA congenital muscular dystrophy (LMNA-CMD).
A research study using data from the UK Facioscapulohumeral muscular dystrophy (FSHD) Patient Registry has been published recently.
Capricor Therapeutics has announced 12-month results from its phase 1/2 clinical trial called HOPE.
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.