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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 disease designation for WVE-210201 – a potential exon skipping drug.Read more

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 of life in people living with Duchenne.Read more

PTC use ataluren trial data to investigate steroids

PTC Therapeutics publishes a paper that uses data from the ataluren trial to investigate the effectiveness of deflazacort and prednisolone.Read more

Clinial hold in Sarepta gene therapy trial

Sarepta Therapeutics has announced that the FDA has issued a Clinical Hold letter for the company’s phase I/II gene therapy trial.Read more

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 departments within a well-known organisation.Read more

EAMS, Raxone and the MHRA

Last week our Director of Campaigns & Strategy, Peter Duffy, attended a meeting at The Medicines and Healthcare products Regulatory Agency (MHRA), as part of the Patient Group Consultative Forum.Read more

Catabasis announces plans for global trial of edasalonexent

Catabasis announces plans to start a global phase 3 trial to test the safety and effectiveness of a potential drug called edasalonexent in people living with Duchenne muscular dystrophy.Read more

Action Duchenne skydive 2018 was amazing

A huge THANK YOU to the 12 amazing fundraisers who jumped out of planes across the country for Duchenne muscular dystrophy this weekend!Read more

Cambridgeshire Dad pushes himself to the limit

A huge thank you and well done to the incredible Alexis Ellison who completed his 24 hour mammoth bike ride - cycling the 9 mile road back and forth from his home town of St Neots to Godmanchester for a whole 24 hours, completely unsupported!Read more

Summit announces results of PhaseOut DMD clinical trial of Ezutromid

Although Ezutromid was safe and well tolerated, after 48 weeks the trial clearly showed that there was no clinical benefit for those who received Ezutromid. As a result, Summit have decided to discontinue the development of Ezutromid.Read more

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 hope could offer similar benefits to steroids with reduced side effects.Read more

HELLO FROM YOUR NEW ACTION DUCHENNE TEAM!

We are dedicated in delivering our vision to you: to have a world where lives are no longer limited by Duchenne or Becker Muscular Dystrophy. Developing effective treatments for all, building a supportive community, and striving for a more inclusive society will always be at the heart of what we do.  Join us in raising the profile of Duchenne, funding pioneering research and clinical trials and educating society on inclusion and disability rights.Read more

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 opinion for Raxone.Read more

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