Raxone (also known as Idebenone) is a drug produced by Santhera Pharmaceuticals, which treats several conditions and has the potential to treat …
Santhera announces idebenone study results
In a press release, Santhera Pharmaceuticals has announced results from the SYROS study, where long-term treatment with idebenone (also known as …
Duchenne Parent Project International Conference
This weekend our Director of Research, Neil Bennett, and our CEO, Shelley Simmonds, are in Rome attending the Duchenne Parent Project International …
Golf Club raise £3,050 in villa raffle
Last weekend, our wonderful supporters Stoke By Nayland Golf Club held their much anticipated raffle draw. The prize was a week in a luxury villa …
Solid Biosciences announce preliminary trial results
Solid Biosciences have announced preliminary data from their phase 1/2 IGNITE-DMD clinical trial that is testing the safety and effectiveness of …
Solid Biosciences announce preliminary trial resultsRead More
Family urge PTC to fight for Translarna approval
PTC Therapeutics had their annual kick-off meeting in Cascais, outside of Lisbon from 28th January to the 1st of February. Cormac Fegan, age 8, and …
Call to action – Powerchair Football in Scotland
An update from our Scottish Advocacy Officer, John Miller: "At a Disability Cross Party meeting on the 27th November 2018, the Secretary of …
HOPE-2 update from Capricor Therapeutics
Following meetings with the FDA (the medicines regulator in the US), Capricor Therapeutics yesterday issued an update yesterday on it’s HOPE-2 …
What your money buys
If you're supporting one of our many Duchenne research projects, here's an idea of where your money goes, and how every penny …
Edasalonexent update from catabasis
Catabasis has announced that the phase 3 trial of edasalonexent is now recruiting at 9 centres across the USA and that regulatory approval for the …
Sarepta completes accelerated approval application for golodirsen
Sarepta therapeutics has today announced that they have completed the submission of a USA application for accelerated approval of golodirsen …
Sarepta completes accelerated approval application for golodirsenRead More
UNITE-DMD project update – Year 1
Gene therapy for Duchenne muscular dystrophy aims to compensate for the lack of dystrophin by transferring a working version of the dystrophin gene …
