June 7, 2019

PTC Receives FDA Approval for Expansion of EMFLAZA® (deflazacort) Labeling to Include Patients 2-5 Years of Age

PTC Therapeutics announced that they have received FDA approval for the expansion of the EMFLAZA® (deflazacort) labeling to include patients 2-5 years of age. Emflaza was first approved by the FDA in February 2017 for…

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May 31, 2019

WATCH: Domagrozumab as a Potential Treatment for Duchenne – Webinar Recording

On May 22, 2019, Pfizer Inc. joined Parent Project Muscular Dystrophy for a webinar to provide the community with an overview of the primary safety and efficacy results from the Phase 2 clinical trial. Additional…

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May 20, 2019

PPMD’s Certified Duchenne Care Center Program – 2018 Impact & Progress

For 25 years, care has been at the heart of everything PPMD does. We strive to ensure that people living with Duchenne are living longer, stronger lives, by helping you access expert healthcare providers, a…

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May 14, 2019

Solid Biosciences Provides Update on IGNITE DMD Status and New Clinical Sites

Solid Biosciences shared an update on the progress of their IGNITE DMD Phase I/II clinical trial for investigational microdystrophin gene transfer, SGT-001. The update includes news about initiating dosing for their higher dose cohort of…

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May 13, 2019

WATCH: Santhera Update to the Duchenne Community – Webinar Recording

On May 8, 2019, Santhera joined Parent Project Muscular Dystrophy for a webinar to provide the community with an update about respiratory health in Duchenne muscular dystrophy and the ongoing SIDEROS clinical trial. If you…

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April 30, 2019

WATCH: Capricor’s Clinical Program – Webinar Recording

On Wednesday, April 24, 2019, Capricor joined Parent Project Muscular Dystrophy for a webinar to update the community on Capricor’s HOPE2 clinical program. If you missed the live event, the recording can be found below….

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April 16, 2019

Wave Life Sciences Announces Suvodirsen Phase 1 Safety and Tolerability Data and Phase 2/3 Clinical Trial Design

Wave Life Sciences shared safety and tolerability data from their Phase 1 clinical trial evaluating investigational suvodirsen (WVE-210201) in patients with Duchenne. The results from this trial support progressing to a Phase 2/3 clinical trial,…

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April 15, 2019

FibroGen Receives U.S. FDA Orphan Drug Designation for Pamrevlumab for the Treatment of Duchenne

FibroGen announced that FDA has granted Orphan Drug Designation for the company’s anti-CTGF antibody, pamrevlumab, for the treatment of people with Duchenne. According to their press release, all 21 non-ambulatory Duchenne patients in their ongoing…

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April 8, 2019

Audentes Therapeutics Announces Expansion of AAV Technology Platform and Pipeline for Duchenne

Audentes Therapeutics announced that it is expanding its scientific platform and beginning development of new AAV-based genetic medicines for Duchenne. Audentes is a leading AAV-based genetic medicines company based in San Francisco. The company is…

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April 5, 2019

Catabasis Shares March 2019 Newsletters

Catabasis shared the latest edition of their community newsletter (also available in Spanish), including an update on their currently recruiting Phase 3 PolarisDMD trial for edasalonexent in Duchenne. Download the Catabasis newsletters: English Spanish

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