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 dystrophin gene (micro-dystrophin) to the leg muscles of people living with Duchenne. The early results are encouraging, but it is too early to say whether the potential treatment will prove to be safe or effective.Read more

CEO attends EURORDIS Summer School

Last week our incoming CEO, Shelley Simmonds, completed a 5 day intensive training programme in Barcelona, having been selected to take part in the 'Expert Patient and Researcher EURORDIS Summer School'.Read more

Solid Biosciences announce FDA have removed clinical hold from their gene therapy trial

Solid Biosciences Announces FDA Removes Clinical Hold on SGT-001 - Activities to Resume Enrollment in IGNITE DMD Phase I/II Clinical Trial are UnderwayRead more

DMD Pathfinders launch new nutrition guide for adults

They have pulled together existing best practice and experiences of adults with Duchenne into a useful resource.Read more

New technology could highlight potential treatments for Duchenne muscular dystrophy

Researchers at Queen Mary University London are investigating a cell based method to potentially increase the understanding of treating Duchenne.Read more

Action Duchenne represent our community at bone protective therapy ENMC workshop

Our DMD Registry Curator, Angela Stringer recently attended the 236th EMNC International Workshop.Read more

Take part in ventilator study and earn £75

Action Duchenne are helping SuAzio to recruit participants. By participating in an interview you will help to support companies with their research relating to Duchenne and will also increase the knowledge of Duchenne across the scientific community.Read more

CHMP Adopts Positive Opinion for the Expansion of the Translarna (ataluren) Label to Include Patients as Young as 2 Years of Age

- European Commission ratification anticipated in coming months - - Approval of the Translarna annual re-assessment also recommended by CHMP -Read more

As Expected, Sarepta Receives Negative CHMP Opinion for EXONDYS (eteplirsen) to Treat Patients with Duchenne in Europe

Sarepta to seek re-examination of the opinion and request that a Scientific Advisory Group (SAG) be convenedRead more

Daiichi Sankyo announces Phase 1/2 clinical trial results

No safety concerns, such as discontinuation or clinically significant adverse events, were observed in the study. The expression of dystrophin protein, the primary endpoint of efficacy, was partially identified, but was not be clearly detected as a whole. However, the secondary endpoint of efficacy, the production of messenger RNA with exon 45 skipping of the dystrophin gene, was found in all patients.Read more

Sarepta collaborate with Invitae to speed up diagnosis of Duchenne

Collaboration aims to speed up diagnosis and expand research into Duchenne.Read more

Thomas Blumire's music to be performed at West Midlands concert

Thomas's uplifting composition "It Couldn't Be Done" will be performed by the Music of Life Foundation on June 20.Read more

Teachers brave the Zipwire!

This weekend saw an amazing group of teachers and supporters from Bruntcliffe Academy take on the world's fastest, and Europe's longest Zipwire.Read more

Solid announces upcoming pre-clinical data presentations

Solid will present new pre-clinical data from SGT-001 at The American Society of Gene and Cell Therapy.Read more

FDA Grants Orphan Drug Designation to Sarconeos

BIOPHYTIS have announced that the US Food and Drug administration (FDA) has granted orphan drug designation to its drug candidate, Sarconeos.Read more

NICE update on Eteplirsen

NICE update on Eteplirsen Following last week’s announcement that Sarepta’s exon skipping drug, Eteplirsen, had received a negative trend vote from the European Medicine Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP).Read more

Solid Biosciences financial results and business update

Company Finalizing Response to FDA Regarding Clinical Hold on SGT-001 Phase I/II Clinical Trial.Read more

Catabasis financial reports and business progress

Edasalonexent significantly slowed Duchenne muscular dystrophy (DMD) disease progression as measured by MRI through one year of treatment. Height and weight through 60 weeks of edasalonexent treatment was on track with standard growth curves for unaffected boys.Read more

Action Duchenne announces changes and new key appointments

The Board of Trustees are today excited to inform the Duchenne and Becker community of three new key staff appointments at our organisation - a new CEO, Director of Research and Director of Campaigns & Strategy - to compliment and strengthen our current team and operations at Action Duchenne.Read more

Wave Life Sciences financial results and update

The global Phase 1 clinical trial, testing WVE-210201 for the treatment of Duchenne muscular dystrophy (DMD) patients amenable to exon 51 skipping, continues to enroll patients. Safety data from the trial are anticipated in the third quarter of 2018.Read more

Changing Places, Changing Lives

"Going to the toilet is a basic human right and Changing Places facilities offer a clean, spacious and private environment to those who need it - having a hoist and changing bench makes a world of difference."Read more

Delay in access to Exondys 51

Last week, at the European Medicine’s Agency (EMA) the Committee for Medicinal Products for Human Use (CHMP) held a hearing looking at whether to grant a regulatory approval for eteplirsen (also known as Exondys 51) in Europe.Read more