Please take a few minutes to complete this short, anonymous online survey to find out about the side effects of steroids that impacts most on quality …
PTC use ataluren trial data to investigate steroids
Last week PTC Therapeutics published a paper demonstrating that over 1 year, deflazacort slowed the decline in the distance boys living with Duchenne …
PTC use ataluren trial data to investigate steroidsRead More
Clinial hold in Sarepta gene therapy trial
Yesterday, Sarepta Therapeutics announced that the FDA has issued a Clinical Hold letter for the company’s phase I/II trial testing the safety and …
Internships
We have been offered a number of paid internships for young people living with Duchenne. The positions are available this summer across a range of …
EAMS, Raxone and the MHRA
Last week our Director of Campaigns & Strategy, Peter Duffy, attended a meeting at The Medicines and Healthcare products Regulatory …
Catabasis announces plans for global trial of edasalonexent
Catabasis has announced plans to start a global phase 3 trial to test the safety and effectiveness of a potential drug called edasalonexent in people …
Catabasis announces plans for global trial of edasalonexentRead More
Action Duchenne skydive 2018 was amazing
A huge THANK YOU to the 12 amazing fundraisers who jumped out of planes across the country for Duchenne muscular dystrophy this weekend! YouTube …
Cambridgeshire Dad pushes himself to the limit
A huge thank you and well done to the incredible Alexis Ellison who completed his 24 hour mammoth bike ride - cycling the 9 mile road back …
Summit announces results of PhaseOut DMD clinical trial of Ezutromid
Earlier today Summit Therapeutics announced the results of the PhaseOut DMD clinical trial of Ezutromid. Although Ezutromid was safe and well …
Summit announces results of PhaseOut DMD clinical trial of EzutromidRead More
ReveraGen BioPharma announces preliminary results of Vamorolone trial
ReveraGen BioPharma has announced encouraging preliminary results of their early stage clinical trial of Vamorolone, a potential drug that researchers …
ReveraGen BioPharma announces preliminary results of Vamorolone trialRead More
HELLO FROM YOUR NEW ACTION DUCHENNE TEAM!
We would like to start the week by saying hello and introducing ourselves as your new Action Duchenne team! We are dedicated in delivering our vision …
Santhera announces renewal of Raxone EAMS
Santhera has announced that the MHRA (the UK’s drug and medicines regulator) has granted a renewal of the Early Access to Medicines Scheme scientific …
