The results of the first clinical trial of vamorolone (which ended earlier this year) in people living with Duchenne were published last week in …
Gene editing restores dystrophin production in dog model of Duchenne
Last week researchers from the United States and London’s Royal Veterinary College published a paper demonstrating that gene editing techniques …
Gene editing restores dystrophin production in dog model of DuchenneRead More
Pfizer to discontinue trials of domagruzumab in Duchenne
We’ve just heard that Pfizer is halting clinical trials of domagrozumab for Duchenne muscular dystrophy after it failed to show effectiveness in …
Pfizer to discontinue trials of domagruzumab in DuchenneRead More
World Duchenne Awareness Day 2018
Tomorrow, 7th September, marks annual Duchenne Awareness Day – a day in which the entire community comes together with one clear mission – …
Wave gain Orphan Drug status for WVE-210201
Wave Life Sciences has announced that the Drug Regulator in the USA – the FDA - has granted both orphan drug designation and rare paediatric …
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
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
Spotlight on Fundraisers July 2018
Here are some of our wonderful fundraisers for this month! …
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 …
Sarepta announce early, preliminary gene therapy trial results
Sarepta therapeutics has announced preliminary results of a gene therapy trial that is using a harmless virus to deliver a small but functional …
Sarepta announce early, preliminary gene therapy trial resultsRead More
