October 27, 2021

Calling All Individuals with Becker Muscular Dystrophy – We Need You!

Do you or a loved one have a diagnosis of Becker muscular dystrophy? If so, please read these important updates which are focused on how we can help individuals with Becker and their families. Becker…

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October 22, 2021

Race Athlete Spotlight: Matthew Colon

Meet Matthew Colon Why did you choose to join the Race to End Duchenne team? Parent Project Muscular Dystrophy has given me hope for my sons future and for that, I am honored to be…

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October 18, 2021

WATCH: SRP-9001 Micro-Dystrophin Gene Therapy Clinical Update (Webinar Recording)

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.*…

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October 14, 2021

The Duchenne Registry has a new survey – Therapy Services!

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…

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October 11, 2021

Sarepta Therapeutics holds SRP-9001 R&D Day

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….

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October 8, 2021

Sarepta SRP-9001 Micro-dystrophin Gene Therapy Update and R&D Day

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…

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October 7, 2021

Dyne Therapeutics Presents New Preclinical Data on the Potential of FORCE Platform to Address the Genetic Basis of Duchenne

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…

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October 6, 2021

PPMD Completes Successful Pilot in Newborn Screening for Duchenne Muscular Dystrophy

PPMD’s Newborn Screening Pilot recently completed two years of ground-breaking recruitment. More than 36,000 families consented to participate and more than 36,000 babies have been screened for Duchenne Muscular Dystrophy in New York State. As…

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October 5, 2021

PPMD releases results of second survey on impact of pandemic on families

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…

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October 4, 2021

Sarepta Therapeutics in partnership with Roche announced the initiation of the EMBARK clinical trial for their investigational gene therapy product SRP-9001

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…

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