November 29, 2021

Support PPMD Tomorrow, on #GivingTuesday

#GivingTuesday is a global generosity movement, bringing together the power of people and organizations to transform their communities and their world. 2021 has once again highlighted  the power of our Duchenne community. Although we came…

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November 24, 2021

This Thanksgiving I am grateful for you

The turkey is prepped, and we are ready to turn on the oven bright and early tomorrow. My emotions are always heightened  during the holidays, but this year it’s even more palpable. The holidays are…

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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 18, 2021

WATCH: Connect with the Experts – Pulmonary Care (October 2021)

During this year’s PPMD Virtual Annual Conference, we hosted a panel titled: A Patient’s Take on Pulmonary Management. We received a large volume of questions during this live session, so many that we were only…

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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 16, 2021

Pediatric Gene Therapy and Medical Ethics (PGTME) holds 2nd Annual Lecture Series

As gene therapy continues to advance through clinical trials, many new concepts, terms and issues arise, including those related to research design, patient autonomy, sibling and family involvement, fairness in access and the quality of…

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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 9, 2021

WATCH: Pediatric COVID-19 Vaccination (ages 5-11) & Duchenne (Webinar Recording)

On November 5, 2021, PPMD invited Drs. Tim Cripe and Tim Franson back for another COVID-19 vaccine webinar to provide updates on the new pediatric approval and what it means for the Duchenne community. Our…

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November 8, 2021

Recap: Recent Research Updates

Welcome to FALL Time flies! Over the past few months, we have seen a number of announcements regarding drug development, changes to clinical trials, and new trials opening or on the horizon. For many, it…

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