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Acceleron announced today that the US FDA has granted Orphan Drug designation to their drug ACE-083 for the treatment of patients with Charcot-Marie-Tooth disease (CMT).
French pharmaceutical company Pharnext has announced that PXT3003 has been granted Fast Track Designation by the US Food and Drug Administration (FDA).
Pharnext is carrying out an observational study for people with Charcot-Marie Tooth disease using a new digital app called CMT&Me.
The US Food and Drug Administration has granted Fast Track Designation to ACE-083 for the treatment of individuals with Charcot-Marie-Tooth Disease (CMT).
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.
Acceleron has announced interim results from its ongoing trial testing ACE-083 in adults with CMT.
A recent preclinical study has identified a potential therapeutic target for Charcot Marie Tooth disease (CMT).
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.
Results from an MDA-supported study show that progressive resistance exercise is safe and can significantly reduce muscle weakness in children with CMT.
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.