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Latest results from Solid gene therapy trial
Solid Biosciences have today released an update on their clinical trial of a gene therapy for Duchenne. The IGNITE DMD trial is an early stage …
Golodirsen gains conditional approval in US
Sarepta Therapeutics has announced that the FDA (the medicines regular in the US) has granted accelerated approval to golodirsen. Golodirsen, or …
Update on UNITE-DMD project
The UNITE-DMD project, which we are co-funding alongside Muscular Dystrophy UK and AFM-Téléthon brings together several strands of research to …
Duchenne Science events back on the road in 2020
We've welcomed over 100 people to our Science on Tour events throughout 2019, these empowering and invaluable sessions have been a huge success and we …
Solid gene therapy trial placed on hold
Solid BioSciences has announced that the IGNITE DMD trial - a Phase I/II clinical trial of the company’s SGT-001 gene therapy - has been placed …
Vamorolone gains Promising Innovative Medicine status
We’re happy to announce that the MHRA (the medicines regulator in the UK) has given Promising Innovative Medicine (or PIM) status to Vamorolone as a …
Vamorolone gains Promising Innovative Medicine statusRead More
Wave gives suvodirsen update
Wave Life Sciences announced earlier this week that Suvodirsen has been granted fast track approval by the Federal Drug Administration (the drug …
Vamorolone trial results published
Earlier this week the results of an early trial of vamorolone in boys with Duchenne have been published in the Neurology scientific journal. …
Ignite DMD update
In a press release yesterday, Solid Biosciences gave a short update on their gene therapy trial called Ignite DMD. Following the decision earlier this …
Sarepta issues clinical trials update
In recent press releases, Sarepta Therapeutics has given updates on their ongoing clinical trial programmes. These include exon skipping and gene …
UNITE-DMD project update – Year 1
Gene therapy for Duchenne muscular dystrophy aims to compensate for the lack of dystrophin by transferring a working version of the dystrophin gene …
