These are exciting times, with fresh news about research developments appearing every week.
Find out about the latest research news below and also visit our Breaking News in Research page.
Joint letter to NICE regarding the suspension of the appraisal process for ridisplamRead more
Our Research Communications Manager, Dr Jenny Sharpe, recently attended the twelfth meeting of the International Myotonic Dystrophy Consortium (IDMC) in Gothenburg, Sweden.
Scientists from Fred Hutchinson Cancer Research Center (USA) have identified a new role for DUX4, the gene that is mutated in FSHD
Capricor Therapeutics has released positive preliminary results from its HOPE-2 Phase II clinical trial testing CAP-1002 in steroid-treated boys and young men with DMD.
The FSH Society International Research Congress was held in France, on 19-20 June 2019
Dynacure has announced that the European Medicines Agency (EMA) has granted Orphan Drug designation to DYN101 for the treatment of patients with centronuclear myopathy (CNM).
Benitec Biopharma has announced delays in the development of its gene therapy to treat OPMD.
The registry is once again live. If you haven’t yet signed up to it, you can do so now, and if you have signed up for it previously, you can go in and update your records.
Pfizer, FibroGen and Catabasis share new data on their clinical trials.
Positive results found from blocking myostatin in a mouse model of OPMD. This demonstrates that treatments not targeting the genetic cause could still be beneficial.
The US FDA has granted approval to Zolgensma, the first gene therapy for SMA. Although the approval process in Europe is separate to the US, it’s a major step forward.