Sarepta therapeutics has announced a further set of preliminary results of a gene therapy trial that is using a harmless virus to deliver a small but …
Sarepta Therapeutics has announced the latest results from their gene therapy trialRead More
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 Therapeutics gene therapy trial to continue
Sarpeta Therapeutics has announced that the Food and Drug Administration (FDA – the drug regulator in the USA) has lifted the Clinical Hold that was …
Sarepta Therapeutics gene therapy trial to continueRead More
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
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
