Filter

Duchenne Parent Project International Conference

This weekend our Director of Research, Neil Bennett, and our CEO, Shelley Simmonds, are in Rome attending the Duchenne Parent Project International Conference.Read more

Golf Club raise £3,050 in villa raffle

Last weekend, our wonderful supporters Stoke By Nayland Golf Club held their much anticipated raffle draw. The prize was a week in a luxury villa in Cyprus, with space for 8 people to enjoy the pool, sun and gorgeous relaxing surroundings.Read more

Solid Biosciences announce preliminary trial results

Solid Biosciences have announced preliminary data from their IGNITE-DMD clinical trial that is testing the safety and effectiveness of an adeno-associated virus (AAV) gene therapy.Read more

Family urge PTC to fight for Translarna approval

Cormac Fegan, age 8, and his family were invited to give a Q&A session to mark the start of the second day of the event to over 150 attendees from all areas of the company covering all geographic areas.Read more

Call to action - Powerchair Football in Scotland

Please join the Cross Party Debate discussing Powerchair football in Scotland on Wednesday the 6th of February 2019 at 5:15pm in Scottish Parliament.Read more

HOPE-2 update from Capricor Therapeutics

Capricor Therapeutics update on it’s HOPE-2 clinical trial of CAP-1002, a potential treatment for Duchenne muscular dystrophy.Read more

What your money buys

If you're supporting one of our many Duchenne research projects, here's an idea of where your money goes, and how every penny counts.Read more

Edasalonexent update from catabasis

Catabasis has announced that the phase 3 trial of edasalonexent is now recruiting at 9 centres across the USA and that regulatory approval for the trial to start in Europe has been received.Read more

Sarepta completes accelerated approval application for golodirsen

Sarepta Therapeutics has completed the submission of a request for accelerated approval for golodirsen in the USARead more

UNITE-DMD project update - Year 1

Update on the progress of the UNITE-DMD programme that we fund jointly with MDUK and AFM-Telefon.Read more

Wave Life Sciences announce phase 1 trial results

Wave Life Sciences has announced results of the phase 1 trial of the WVE-210201 – a potential drug that induces skipping of exon 51 of the dystrophin geneRead more

ENMC Workshop in Amsterdam

Our Director of Research, Neil attended the 238th ENMC workshop.Read more

Action Duchenne present parent's perspective to healthcare professionals

We as a charity relish every opportunity to advocate life with Duchenne to professionals in the Healthcare field.Read more

Santhera acquires rights to vamorolone

Santhera has announced that they have acquired the rights to vamorolone - a drug that has been developed and is currently being tested in trials by Reveragen.Read more

Sarepta Therapeutics has announced the latest results from their gene therapy trial

Sarepta therapeutics has announced a further set of preliminary results from their early-stage gene therapy trialRead more

Catabasis starts Phase 3 trial of edasalonexent

Catabasis announce that a Phase 3 trial of edasalonexent has started, with trial centres to open shortly – first in the USA and then in Europe.Read more

Sarepta Therapeutics gene therapy trial to continue

The FDA has lifted the Clinical Hold that was issued to Sarepta Therapeutics phase I/II gene therapy trialRead more

Sarepta receives negative opinion following CHMP review of eteplirsen

Sarepta has received a negative opinion of eteplirsen following a second review by the European Medicine's Agency Committee for Medicinal Products for Human Use (CHMP).Read more

Vamorolone trial results published

The results of the first clinical trial of vamorolone (which finished in 2016) in people living with Duchenne were published last weekRead more

Berardelli family fundraise at Lochaber Agricultural Show

Elspeth and Paolo have been dedicated fundraisers for many years now following their son Georgio's diagnosis with Duchenne.Read more

Gene editing restores dystrophin production in dog model of Duchenne

Researchers from the United States and London’s Royal Veterinary College have demonstrated that gene editing techniques can restore dystrophin production in a dog model of Duchenne.Read 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 – for our families and raising the profile of Duchenne muscular dystrophy to a wider audience.Read more

Pfizer to discontinue trials of domagruzumab in Duchenne

Pfizer has announced that trials of domagrozumab are to be discontinued after it failed to show effectiveness in a phase 2 clinical trial.Read more