News

At Duchenne Patient Academy, DMD and BMD patient advocates receive training to build a strong base for current and future global advocacy.

The post Applications open for Duchenne Patient Academy 2021 appeared first on World Duchenne.

Data shows Translarna delays loss of ambulation by more than five years in nonsense DMD boys.

The post STRIDE data shows Translarna delays loss of ambulation by more than five years in nonsense DMD boys appeared first on World Duchenne.

The Akari Organization supports low-income Hispanic families with people affected by Duchenne in San Antonio, Texas. 

The post The Akari Foundation joins World Duchenne Organization appeared first on World Duchenne.

The recording from the online event 'Adult Life & Duchenne' is now available for rewatching. Interpretation available in multiple languages.

The post ONLINE EVENT: ADULT LIFE & DUCHENNE appeared first on World Duchenne.

All World Duchenne Awareness Day 2021 events involving thousands of people across the world, under the theme ‘Adult Life & Duchenne’.

The post World Duchenne Awareness Day highlights a year-round effort to improve adult life in Duchenne appeared first on World Duchenne.

The Journal of Neuromuscular Diseases published the consensus guidelines for adult Duchenne care.

The post Consensus Guidelines for Adult Duchenne Care appeared first on World Duchenne.

During the second FAIR Data Meeting for Duchenne, 120 participants from 22 countries discussed how they could drive FAIR data efforts to facilitate research and healthcare.

The post How Patient Organizations Can Drive FAIR Data Efforts to Facilitate Research and Health Care appeared first on World Duchenne.

Adam Peska from the Czech Republic won a gold medal in individual boccia BC3 at the 2020 Summer Paralympics in Tokyo, Japan.

The post Adam Peska to win gold medal in Boccia at Tokyo Paralympics appeared first on World Duchenne.

Today, Philip Jönsson won the gold medal in the mixed 10m air rifle standing SH2 event at the 2020 Summer Paralympics held in Tokyo, Japan.

The post Philip Jönsson first person with Duchenne MD to win gold at Paralympics appeared first on World Duchenne.

The aim of this ENMC workshop were to raise priority and need for clinical and research focus on DMD female carriers. Report now available.

The post ENMC workshop to refine recommendations for female carriers appeared first on World Duchenne.

Last week, Parent Project Muscular Dystrophy was joined by Sarepta Therapeutics for a webinar to discuss the details of the EMBARK clinical trial (SRP-9001-301). If you missed the live event, the recording can be found below.*...

The post WATCH: SRP-9001 Micro-Dystrophin Gene Therapy Clinical Update (Webinar Recording) appeared first on Parent Project Muscular Dystrophy.

We know that therapy services such as physical, occupational, and speech therapy are helpful to people with Duchenne and Becker throughout their lives.   The new Therapy Services survey was developed to better understand who is...

The post The Duchenne Registry has a new survey – Therapy Services! appeared first on Parent Project Muscular Dystrophy.

This morning, Sarepta Therapeutics held SRP-9001 Micro-dystrophin R&D Day, during which Sarepta shared information from all their trials involving SRP-9001.  New data from the 103 Study of SRP-9001 (ENDEAVOR) using their commercial material was presented....

The post Sarepta Therapeutics holds SRP-9001 R&D Day appeared first on Parent Project Muscular Dystrophy.

On October 4th, Sarepta Therapeutics announced the initiation, in partnership with Roche, of a pivotal study of SRP-9001-301 (rAAVrh74.MHCK7.micro-dystrophin) also known as EMBARK.  Simultaneously, the company announced plans to host a community webinar with PPMD...

The post Sarepta SRP-9001 Micro-dystrophin Gene Therapy Update and R&D Day appeared first on Parent Project Muscular Dystrophy.

Dyne Therapeutics presents new preclinical data on the potential of FORCE platform to address the genetic basis of Duchenne. Dyne Therapeutics recently released new preclinical data from their Duchenne muscular dystrophy program, which was initially...

The post Dyne Therapeutics Presents New Preclinical Data on the Potential of FORCE Platform to Address the Genetic Basis of Duchenne appeared first on Parent Project Muscular Dystrophy.

PPMD’s Newborn Screening Pilot recently completed two years of ground-breaking recruitment. More than 37,000 families consented to participate and more than 37,000 babies have been screened for Duchenne Muscular Dystrophy in New York State. As...

The post PPMD Completes Successful Pilot in Newborn Screening for Duchenne Muscular Dystrophy appeared first on Parent Project Muscular Dystrophy.

Since the start of the COVID-19 pandemic, Parent Project Muscular Dystrophy (PPMD) has surveyed Duchenne and Becker families to better understand how the patient experience with care, clinical trials, and access to approved therapies has...

The post PPMD releases results of second survey on impact of pandemic on families appeared first on Parent Project Muscular Dystrophy.

Sarepta Therapeutics, in partnership with Roche, has announced the initiation of study SRP-9001-301, also known as EMBARK, a pivotal study of SRP-9001 (rAAVrh74.MHCK7. micro-dystrophin) for the treatment of Duchenne muscular dystrophy. It is important to...

The post Sarepta Therapeutics in partnership with Roche announced the initiation of the EMBARK clinical trial for their investigational gene therapy product SRP-9001 appeared first on Parent Project Muscular Dystrophy.

9. Duchenne Gene Therapy Update
1 October 2021, 1:27 pm

As many in the community are aware, on Monday, September 28, Pfizer released a statement to the public announcing a change in their Phase III gene therapy trial for Duchenne. We applaud their efforts to...

The post Duchenne Gene Therapy Update appeared first on Parent Project Muscular Dystrophy.

  This morning, Wave Life Sciences Ltd. announced the initiation of dosing in a Phase 1b/2a clinical trial expecting to enroll 15 boys, evaluating investigational WVE-N531 as a treatment for boys with Duchenne muscular dystrophy...

The post Wave Life Sciences Announces Initiation of Dosing in Phase 1b/2a Duchenne Clinical Trial of WVE-N531 Starting in Canada and the UK appeared first on Parent Project Muscular Dystrophy.