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
Vamorolone trial results published
The results of the first clinical trial of vamorolone (which ended earlier this year) in people living with Duchenne were published last week in …
Berardelli family fundraise at Lochaber Agricultural Show
Beneath the spectacular backdrop of Ben Nevis, the Lochaber Agricultural Show is an amazing celebration of what rural Lochaber has to offer. …
Berardelli family fundraise at Lochaber Agricultural ShowRead More
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 …
Take part in 5 minute online corticosteroids study
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 …
