October 4, 2019

PTC Announces New Real-World Analysis Demonstrating Translarna Slows Disease Progression in Duchenne

As PPMD reported in our World Muscle Society recap yesterday, PTC Therapeutics has announced that data from their STRIDE registry demonstrate that Translarna preserves lung function in children and adolescents compared with a matched cohort…

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October 3, 2019

2019 World Muscle Society Update – Day 1

This week, PPMD’s Abby Bronson, SVP of Research Strategy and Eric Camino, PhD, Director of Research and Clinical Innovation are attending the annual World Muscle Society (WMS) meeting, this year in Copenhagen, Denmark. Below, Abby…

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October 2, 2019

NS Pharma Completes Rolling NDA Submission to FDA for NS-065/NCNP-01 (viltolarsen)

We are pleased to learn that NS Pharma has completed the submission of its rolling New Drug Application (NDA) to the FDA for NS-065/NCNP-01 (viltolarsen), which is under development for the treatment of Duchenne amenable…

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October 1, 2019

Catabasis Completes Phase 3 PolarisDMD Trial Enrollment

Catabasis announced that the Phase 3 PolarisDMD trial for edasalonexent in Duchenne has completed enrollment and exceeded the target enrollment. PPMD is proud that The Duchenne Registry was able to play a role in helping…

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September 25, 2019

Antisense Therapeutics Announces Positive Preliminary Results from ATL1102 for Duchenne Phase II Trial

Antisense Therapeutics has announced that a review of the preliminary data from the 6 patients who have completed their 24 weeks of dosing in the Phase II clinical trial of the company’s immunomodulatory therapy, ATL1102…

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September 25, 2019

Santhera Shares Fall 2019 Community Update

Santhera has shared a community update on the company’s Duchenne drug development programs and their partnership with ReveraGen. Click here to download.

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September 23, 2019

Italfarmaco Shares Fall 2019 Newsletter

Italfarmaco has shared the Fall 2019 issue of their newsletter, including updates on the Givinostat Development Program. Click here to download.

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September 18, 2019

Recap of PPMD’s Duchenne Platform Trial Community Meeting

On Monday September 9, PPMD along with I-ACT for Children and the Duchenne Regulatory Science Consortium (DRSC) held an open meeting inviting all stakeholders (patients, families, industry sponsors, and regulators) in Duchenne to learn and…

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September 16, 2019

Wave Life Sciences Announces Fast Track Designation from U.S. FDA for Suvodirsen

Wave Life Sciences shared today that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to suvodirsen for the treatment of Duchenne in patients amenable to exon 51 skipping. Fast Track designation…

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September 16, 2019

PPMD Unveils Care Materials & Remote Tour Stop in SPANISH

Each year during Duchenne Action Month, PPMD reminds our families to download our comprehensive care materials, including critical emergency information. This information is especially important as kids head back to school to help educate school staff….

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