Solid Biosciences have announced that the FDA has given approval for the company to restart the phase I/II IGNITE DMD clinical trial. The clinical …
Solid Biosciences announce FDA have removed clinical hold from their gene therapy trialRead More
New technology could highlight potential treatments for Duchenne muscular dystrophy
Researchers from Queen Mary University of London have developed new cell-based technologies which could improve understanding of the muscle-wasting …
New technology could highlight potential treatments for Duchenne muscular dystrophyRead More
As Expected, Sarepta Receives Negative CHMP Opinion for EXONDYS (eteplirsen) to Treat Patients with Duchenne in Europe
Sarepta will seek re-examination of the opinion and request that a Scientific Advisory Group (SAG) be convened Sarepta have announced that the …
Daiichi Sankyo announces Phase 1/2 clinical trial results
Daiichi Sankyo have announced the top-line results of the Phase 1/2 clinical trial in Japan of DS-5141), which Daiichi Sankyo is jointly …
Daiichi Sankyo announces Phase 1/2 clinical trial resultsRead More
Spotlight on Fundraisers June 2018
Here are some of our wonderful fundraisers for this month! Two new skydivers' pages https://www.justgiving.com/fundraising/tracy-tuck1 …
Support for the Shippey family at their annual Golf Day
96 amazing golfers and a further 34 evening guests gathered to support the wonderful Shippey family at their second annual Cure4George charity golf …
Support for the Shippey family at their annual Golf DayRead More
Sarepta collaborate with Invitae to speed up diagnosis of Duchenne
Muscular Dystrophy News Today have reported that Sarepta Therapeutics and Invitae, a company specialising in genetic diagnostics, are …
Sarepta collaborate with Invitae to speed up diagnosis of DuchenneRead More
Thomas Blumire’s music to be performed at West Midlands concert
Music of Life Foundation is a small charity that uses music-making to advance education and improve the health and wellbeing of disabled …
Thomas Blumire’s music to be performed at West Midlands concertRead More
NICE update on Eteplirsen
Following last week’s announcement that Sarepta’s exon skipping drug, Eteplirsen, had received a negative trend vote from the …
Action Duchenne announces changes and new key appointments
At Action Duchenne we aim to do our utmost to communicate the many promising Duchenne and Becker Muscular Dystrophy research projects and …
Action Duchenne announces changes and new key appointmentsRead More
Changing Places, Changing Lives
This Friday, 11th May, one of our Trustees, Shelley, will be taking part in a campaign to raise awareness of the need for more Changing Places …
Delay in access to Exondys 51
Last week, at the European Medicine’s Agency (EMA) the Committee for Medicinal Products for Human Use (CHMP) held a hearing …
