News

During the Duchenne Care Conference, top experts share their experience on specific topics of Duchenne (and Becker) care.

The post Duchenne Care Conference 2021 successfully concluded appeared first on World Duchenne.

Duchenne Ukraine opens an international children's drawing competition to discover and celebrate talented children living with Duchenne.

The post Duchenne Ukraine announces international children’s drawing competition appeared first on World Duchenne.

3. Dr Imelda de Groot Award
3 June 2021, 7:36 pm

During the Duchenne Care Conference, taking place June 1-4 online, the Dr Imelda de Groot Award was announced. The Award was revealed by Elizabeth Vroom, chair of the World Duchenne Organization.

The post Dr Imelda de Groot Award appeared first on World Duchenne.

Share4Rare shares the findings and outputs of their project, setting the base for the next generation of data sharing in rare diseases.

The post Share4Rare: Setting the path for next generation data sharing in rare diseases appeared first on World Duchenne.

DMD Care Association provides support for children and families affected by Duchenne in Romania. They are World Duchenne Organization member.

The post DMD Care Association Romania joins World Duchenne Organization appeared first on World Duchenne.

On World Duchenne Awareness Day, we raise awareness for Duchenne and Becker Muscular Dystrophy. The 2021 theme is Adult Life & Duchenne.

The post World Duchenne Awareness Day 2021 theme announcement appeared first on World Duchenne.

In 1987, a small group of parents regularly met at their clinic appointments. Now, they join the global Duchenne family support community.

The post Duchenne Family Support Group joins WDO appeared first on World Duchenne.

Online Duchenne Care Conference June 1 - 4 for WDO Members where global experts share their knowledge on specific care topics.

The post Duchenne Care Conference 2021 appeared first on World Duchenne.

The World Duchenne Organization starting a working group to create tools and resources to advocate for newborn screening in Duchenne.

The post Working Group for Newborn Screening in Duchenne appeared first on World Duchenne.

Online training course for young patient advocates to receive training and guidance to set up their own patient advocacy effort.

The post Summer Training Course for Young Patient Advocates (STYPA) appeared first on World Duchenne.

Parent Project Muscular Dystrophy (PPMD) and Jesse’s Journey today announced a collaborative research award of $172,000 (CAD) in support of a two-year Clinical Fellowship in Duchenne Endocrinology and Bone Fragility. The award will sponsor the fellowship of...

The post PPMD & Jesse’s Journey Award $172,000 (CAD) Clinical Fellowship in Duchenne Endocrinology & Bone Fragility appeared first on Parent Project Muscular Dystrophy.

PPMD’s 2021 Virtual Annual Conference – Our Hub is Live! If you haven’t registered for PPMD’s 2021 Virtual Annual Conference — now’s the time to do so! Our Conference Hub is now open! This is your opportunity...

The post PPMD’s Virtual Conference Hub is Live! appeared first on Parent Project Muscular Dystrophy.

WATCH: SRP-9001 Micro-Dystrophin Gene Therapy Clinical Update Parent Project Muscular Dystrophy was joined by Sarepta Therapeutics for a webinar update on June 2, 2021. Sarepta was invited to present recent updates from their ongoing clinical...

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

PPMD’s Rachel Schrader and Mena Scavina recently moderated a fantastic Connect with the Experts: Navigating the College Campus with Duchenne webinar. They led a dynamic conversation around what the college experience may look like for...

The post WATCH: Connect with the Experts – Navigating the College Campus with Duchenne appeared first on Parent Project Muscular Dystrophy.

Santhera and ReveraGen today announced positive and statistically significant results from the 24-week readout of the VISION-DMD study of vamorolone in Duchenne. The companies report that study demonstrated strong efficacy across primary and secondary endpoints...

The post Santhera & ReveraGen Announce Positive & Statistically Significant Topline Results in VISION-DMD Study appeared first on Parent Project Muscular Dystrophy.

Astellas Pharma joined PPMD for a webinar on May 19, 2021 to provide an update on the company’s ASP0367 development program. If you missed the live event, the recording can be found below.    ...

The post WATCH: ASP0367 Development Program Update (Webinar Recording) appeared first on Parent Project Muscular Dystrophy.

Sarepta Therapeutics this morning announced positive 12-week expression and safety results from the first 11 participants enrolled in Study SRP-9001-103, an open-label study known as ENDEAVOR being conducted in partnership with Roche. SRP-9001 demonstrated robust...

The post Sarepta Announces Positive 12-Week Expression and Safety Results from Study SRP-9001-103 (ENDEAVOR) appeared first on Parent Project Muscular Dystrophy.

Patient experience data is a critical tool for advancing drug development in Duchenne and for regulatory decision making. Steroids have been part of care standards for over two decades, however, there is a lack of...

The post Take Part in the Duchenne Steroid Patient Experience Survey appeared first on Parent Project Muscular Dystrophy.

Solid Biosciences has shared an update with the community this morning, including a dosing update in the IGNITE DMD Phase I/II clinical trial, promising long-term biopsy data from prior patients dosed with SGT-001 in the...

The post Solid Provides IGNITE DMD Phase I/II Clinical Trial and Pipeline Update appeared first on Parent Project Muscular Dystrophy.

NS Pharma has announced that they will be presenting new, long-term efficacy and safety data from the open-label extension of a Phase 2 study of VILTEPSO® (viltolarsen) at PPMD’s 2021 Virtual Annual Conference next month....

The post NS Pharma Announces VILTEPSO® Long-Term Trial Data to be Presented at PPMD’s 2021 Virtual Annual Conference appeared first on Parent Project Muscular Dystrophy.