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
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
Wave Life Sciences announce phase 1 trial results
In a press release published earlier, Wave Life Sciences has announced positive results of the phase 1 trial of the WVE-210201 – a potential …
Harry Hill’s ‘Comedians Sing Christmas Songs’ for Action Duchenne was a hit!
A huge thank you to our patron, Harry Hill, who hosted one of Action Duchenne's biggest ever events last night. 'Comedians Sing The Christmas Hits' …
Harry Hill’s ‘Comedians Sing Christmas Songs’ for Action Duchenne was a hit!Read More
ENMC Workshop in Amsterdam
ENMC has a unique concept of facilitating and organizing workshops for experts in the field of neuromuscular conditions. Every month a group of 20 - …
Santhera acquires rights to vamorolone
Santhera has announced that they have acquired the rights to vamorolone - a potential drug that has been developed and is currently being tested in …
Comedians sing the Christmas hits!
It's gonna be one unholy night of fun! We are delighted to announce that on 4th December 2018, our patron Harry Hill will be hosting a comedy …
Catabasis starts Phase 3 trial of edasalonexent
Earlier this week Catabasis announced that a Phase 3 trial of edasalonexent in Duchenne has started, with trial centres to open shortly – first …
Sarepta receives negative opinion following CHMP review of eteplirsen
We've just heard the disappointing news that Sarepta has received a negative opinion of eteplirsen following a second review by the European …
Sarepta receives negative opinion following CHMP review of eteplirsenRead More
