Nusinersen approved for SMA Types 1, 2 and 3Read more
Australian biotechnology company, Benitec Biopharma, recently published pre-clinical results for its OPMD gene therapy programme in the scientific journal, Nature Communications.
Researchers at the University College London (UCL) Institute of Neurology have discovered developmental problems in a mouse model of Charcot Marie Tooth disease (CMT) type 2D.
The aim of the SKIP-NMD project is to develop a potential treatment for Duchenne muscular dystrophy by testing SRP-4053, an exon skipping drug produced by Sarepta Therapeutics.
Acceleron Pharmceuticals has recently announced plans for a phase 2 trial testing ACE-083 in people with Charcot Marie Tooth disease (CMT).
The 2017 UK Neuromuscular Translational Research Conference was jointly organised by Muscular Dystrophy UK and the MRC Centre for Neuromuscular Diseases.
Summit Therapeutics has released an update on its utrophin modulation development programme.
AveXis has released results from its phase 1 trial testing gene therapy in 15 children with SMA type 1.
The US Food and Drug Administration (FDA) has recently agreed to review PTC Therapeutics’ licensing application for Duchenne drug, Translarna.
First HFEA licence granted
The European Medicines Agency (EMA) has granted Orphan Drug Designation to Resolaris (ATYR1940) for the treatment of all types of limb girdle muscular dystrophy (LGMD).