March 19, 2020

Sarepta Therapeutics Shares Community Update on COVID-19

Sarepta Therapeutics has provided the community with an update addressing questions from the community regarding COVID-19, including updates on current clinical trials and accessing approved therapies. Read Sarepta’s Update March 19, 2020 Dear Duchenne and…

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March 18, 2020

Catabasis Shares Update on Edasalonexent and COVID-19

Catabasis Pharmaceuticals has shared an update responding to questions about edasalonexent and COVID-19. To date, Catabasis has not identified any safety concerns related to COVID-19, and they continue to monitor the trial for the safety…

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March 16, 2020

PTC Shares Update on EMFLAZA Availability During Coronavirus

PTC Therapeutics has shared a statement on EMFLAZA availability during the coronavirus (COVID-19) crisis, stating that the supply of EMFLAZA will not be affected by the coronavirus (COVID-19). PTC reports that they are closely monitoring…

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March 12, 2020

Solid Biosciences Provides Letter to the Duchenne Community: Data and IGNITE DMD Clinical Trial Update

Solid Biosciences Inc. provided an update on the IGNITE DMD phase I/II microdystrophin gene therapy clinical program. Solid shares that biomarker data from all three patients dosed in the 2E14 vg/kg cohort of IGNITE DMD…

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March 9, 2020

NS Pharma Announces Launch of Expanded Access Program for Viltolarsen

NS Pharma, Inc. has announced the launch of the viltolarsen Expanded Access Program in the United States for patients with Duchenne muscular dystrophy who are amenable to exon 53 skipping therapy and meet other eligibility…

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March 6, 2020

WATCH: Teen & Young Adult Trial Participation in Duchenne – Industry & Community Perspectives – Webinar Recording

On Wednesday, March 4, 2020, Santhera Pharmaceuticals joined PPMD for a webinar to discuss teen and young adult clinical trial participation. We heard from Santhera about lessons learned by conducting one of the largest trials…

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February 28, 2020

Pfizer Shares Additional Information About PF-06939926 Gene Therapy Trial

Pfizer has provided additional information about their PF-06939926 gene therapy trial. PPMD will continue to share information on this trial as it becomes available. Read Pfizer’s community letter: On Monday, February 24th, 2020, the National…

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February 26, 2020

Catabasis Shares February 2020 Newsletter

Catabasis shares the latest edition of their community newsletter, including an update on edasalonexent, as we approach Rare Disease Day. Click here to download (PDF).

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February 25, 2020

Pfizer Announces Recruitment Information for PF-06939926 Gene Therapy Trial

Yesterday the information for Pfizer’s Phase 3, randomized, double-blind, placebo-controlled study of PF-06939926, exploring the safety and efficacy of gene therapy in patients with Duchenne was posted to clinicaltrials.gov. This will be the largest cohort of patients…

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February 10, 2020

NS Pharma Announces NDA Accepted for Filing by the FDA for Antisense Oligonucleotide Viltolarsen

PPMD is thrilled to learn from Nippon Shinyaku Co., Ltd. and NS Pharma, Inc. that the FDA has filed a New Drug Application (NDA) for their antisense oligonucleotide drug NS-065/NCNP-01 (viltolarsen). Viltolarsen is a drug…

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