December 3, 2021

Dyne Therapeutics Announced Submission of IND Application to Initiate Clinical Trial of DYNE-251

Dyne Therapeutics, Inc. announced yesterday the submission of an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) to initiate a clinical trial of DYNE-251 in patients with Duchenne muscular dystrophy…

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November 23, 2021

Santhera and ReveraGen Announce Positive Topline Results with Vamorolone after Completion of the VISION-DMD Study

PPMD is pleased to share the announcement from Santhera Pharmaceuticals and ReveraGen BioPharma, Inc., of new topline results after completion of the VISION-DMD study at week 48. VISION-DMD was a pivotal double-blind Phase 2b study…

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November 22, 2021

REGENXBIO Announce Orphan Drug Designation Granted to RGX-202, a Novel Gene Therapy Candidate for Duchenne

PPMD is pleased to share that REGENXBIO Inc. announced this morning that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation for RGX-202, a  gene therapy approach for the treatment of Duchenne muscular…

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November 17, 2021

Win a NEW Nintendo Switch OLED!

For all of you gamers and parents of gamers, The Duchenne Registry is excited to announce we will be giving away a brand new, very hard to find, very fun to play Nintendo Switch OLED…

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November 10, 2021

BioMarin shares clinical development update

Late last month BioMarin announced a new pre-clinical candidate for Duchenne, BMN 351. BMN 351 is a third-generation antisense oligonucleotide therapy under investigation for exon 51 skipping that is designed to target a distinct site…

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November 2, 2021

Pfizer’s Phase 3 Gene Therapy Trial, CIFFREO: Community Questions Answered

Gene Therapy is on everyone’s mind these days. We wonder about opportunities, clinical sites, neutralizing antibodies and most recently details of a small number of Serious Adverse Events (SAE) and what these learnings might mean…

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October 28, 2021

Edgewise Therapeutics shares positive topline results from EDG-5506 Phase 1 Study and doses first Becker patients

Edgewise Therapeutics, Inc. announced today positive topline results from the Multiple Ascending Dose (MAD) portion of a first-in-human Phase 1 clinical trial assessing the safety and tolerability of escalating doses of EDG-5506, an orally administered…

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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 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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