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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February 3, 2020

PTC Announces Publication of Data Showing Translarna Significantly Preserves Ability to Walk for Longer

PPMD is excited to learn that PTC Therapeutics announced real-world data showing that boys with nonsense mutation Duchenne treated with Translarna™ (ataluren) and standard of care, preserved the ability to walk for years longer than…

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January 21, 2020

Santhera Shares Winter 2020 Community Update

Santhera Pharmaceuticals shared a letter with the community, emphasizing that their main focus in the U.S. right now is to bring the SIDEROS clinical trial to full enrollment. Learn more about this clinical trial, including…

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January 15, 2020

WATCH: Update on Investigational Suvodirsen in Duchenne Muscular Dystrophy – Webinar Recording

On December 20, 2019, Parent Project Muscular Dystrophy (PPMD) and World Duchenne Organization (WDO) hosted an informational webinar with the Wave Life Sciences team to discuss their decision to discontinue development of suvodirsen for patients…

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January 15, 2020

PPMD’s Duchenne Drug Development Roundtable Creates Clinical Trial Harmonization Guidances

PPMD is excited to share the recently developed Clinical Trial Harmonization Guidances. These guidances are the result of PPMD’s Duchenne Drug Development Roundtable working collaboratively in the pre-competitive space to encourage better consistency across companies…

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January 8, 2020

Italfarmaco Shares Winter 2020 Newsletter

Italfarmaco has shared the Winter 2020 issue of their newsletter, including updates on the Givinostat Development Program. Click here to download.

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January 8, 2020

Catabasis Shares January 2020 Newsletter

Catabasis shares the latest edition of their community newsletter, including information about the Phase 3 PolarisDMD trial enrolling the expected patient population and a partnership with our friends at Duchenne UK to evaluate edasalonexent in…

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December 23, 2019

Sarepta Announces Partnership with Roche Outside the U.S. for its Investigational Micro-dystrophin Gene Therapy

Sarepta Therapeutics, Inc. today announced that Sarepta and Roche have entered into a licensing agreement providing Roche exclusive commercial rights to SRP-9001 (AAVrh74.MHCK7.micro-dystrophin), Sarepta’s investigational gene therapy for Duchenne muscular dystrophy, outside the United States….

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