Early data shows Eteplirsen tolerated safely in young infant with Duchenne

Early data shows Eteplirsen tolerated safely in young infant with DuchenneRead more

Capricor announces repeat dosing of CAP-1002 increases exercise performance in Duchenne mouse

Capricor Announces New Pre-Clinical Study Finds Repeat Doses of CAP-1002 Lead to Enhanced Exercise Capacity in Duchenne Muscular Dystrophy Disease ModelRead more

Summit Completes Dosing of Ezutromid in PhaseOut DMD Clinical Trial

Summit Therapeutics announces the completion of ezutromid dosing in patients with Duchenne muscular dystrophy ('DMD') for the full 48-week PhaseOut DMD clinical trial. Top-line data from the full trial continue to be expected in the third quarter of 2018Read more

Solid address Duchenne community with update on IGNITE DMD Clinical Trial

Solid Biosciences have written to the Duchenne community to provide an update on their IGNITE DMD clinical trial.Read more

Pfizer doses first patient using investigational mini-dystrophin gene therapy

PF-06939926 is an investigational, recombinant AAV9 capsid carrying a truncated or shortened version of the human dystrophin gene (mini-dystrophin) under the control of a human muscle specific promotor. The AAV9 capsid was chosen as the delivery mechanism because of its potential to target muscle tissue.Read more

Wave enter into collaboration with Deep Genomics to expand research programme

Wave Life Sciences and Deep Genomics Form Collaboration to Discover Novel Therapies for Genetic Neuromuscular DisordersRead more

VISION-DMD newsletter launched at Phase 2 of clinical trial of Vamorolone

Action Duchenne supported the preclinical funding and Phase 1 clinical trials of this innovative steroid-like intervention on Duchenne muscular dystrophy.Read more

Give your valuable experiences of caring and supporting a young person with Duchenne

The Ulster University have approached Action Duchenne and our wonderful community to take part in an important research study which could benefit the shaping of future services for families.Read more

'Together for short lives' launches new transition checklist

‘A checklist to a good transition’ is designed for seriously ill young people to assess how well agencies involved in their education and care are working together to enable a smooth transition.Read more

Adelphi Values would like to interview people from the Duchenne community

Action Duchenne are helping Adelphi Values recruiting people from the Duchenne community, to interview for their research studyRead more

Bone Health in Duchenne muscular dystrophy

Your views matter about bone health. There’s still time to take part in an important survey to improve bone health in Duchenne.Read more

ReveraGen receives FDA fast track designation for Vamorolone

This designation can speed the review of efficacy and safety data for vamorolone in boys living with Duchenne, potentially leading to more rapid regulatory approval.Read more

Translarna Managed Access Agreement frequently asked questions

We have worked with Muscular Dystrophy UK to create a Question and Answer document about Translarna and the Managed Access Agreement.Read more

Papworth Trust's Disability in the UK: Facts and Figures 2018

Did you realise that disabled people are TWICE as likely to be unemployed than non-disabled people? Are you aware that there are currently 1.8 million disabled people with unmet housing needs or that disabled people between the ages of 18-65 represent one third of social care users?Read more

Ethics and Social Science – a community in partnership

Last month the Ethics and Social Science Genomics England Clinical Interpretation Partnership (GeCIP) held its first national community meeting in London.Read more

Wave Life Sciences financial results and business update

Wave Life Sciences Reports Fourth Quarter and Full-Year 2017 Financial Results and Provides Business UpdateRead more

You have an important voice - make it heard in the discussion about Raxone and the EAMS

Provide evidence to the MHRA about Raxone and the unmet needs of people living with DuchenneRead more

Summit opens enrolment for people who have taken part in previous Ezutromid trials

Today, Summit announced that they have opened enrolment in PhaseOut DMD to an additional group: those who have previously participated in Phase 1 clinical trials of ezutromid, but did not meet the entry criteria for the main trial.Read more

Antisense receive approval for Phase II trial in Australia

ATL1102 is an antisense inhibitor of CD49d, a subunit of VLA-4 (Very Late Antigen-4).Read more

Recognising Neuromuscular Disorders eLearning module is now live

E-learning resources support paediatricians and other child health professionals to achieve the essential competences in a number of important areas. These can be used to support your training and continuing professional development (CPD).Read more

Shelley Simmonds joined Findacure on the radio to celebrate Rare Disease Day

One of our wonderful Trustees, Shelley Simmonds joined Findacure on Cambridge 105 to celebrate Rare Disease Day 2018.Read more

Action Duchenne funded Sc-OT-DMD study recruited 91 people

Up to 40% of boys lose ambulation earlier after the first fracture, so research into this area is important to allow us to find better ways to treat fragile bones in DMD.Read more

PTC Therapeutics marks Rare Disease Day 2018 with Duchenne and Me app

PTC Therapeutics marks Rare Disease Day 2018 by celebrating 150 year anniversary since Duchenne muscular dystrophy was first describedRead more