February 7, 2019

Solid Biosciences Announces Intention to Dose Escalate in IGNITE DMD Clinical Trial

Solid Biosciences announced today plans to move to a higher dose of SGT-001 in IGNITE DMD, their Phase 1/2 clinical trial evaluating SGT-001 microdystrophin gene therapy as a potential treatment for Duchenne. This decision is…

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February 6, 2019

NS Pharma Begins Rolling NDA Submission to FDA for Viltolarsen (NS-065/NCNP-01)

PPMD is pleased to learn that NS Pharma has initiated submission of a rolling New Drug Application (NDA) seeking accelerated approval for viltolarsen (NS-065/NCNP-01), an investigational drug for the treatment of Duchenne in patients amenable…

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February 6, 2019

Update from the 2nd Annual Duchenne Healthcare Professionals’ Summit

Last month, PPMD hosted the second annual Duchenne Healthcare Professionals Summit. The intent of the Summit was to bring together providers from Certified Duchenne Care Centers and other centers interested in participation in the CDCC…

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February 5, 2019

PPMD to Host Gene Therapy Policy Forum on March 4, 2019

PPMD is excited to announce that this year’s action-packed Advocacy Conference, March 3-5 2019 will include a special Gene Therapy Policy Forum on Monday, March 4 from 9am to 3pm at The Mayflower Hotel in D.C….

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February 1, 2019

Register Today for PPMD’s 2019 Advocacy Conference!

The PPMD Advocacy Conference registration is live! Join us March 3-5 in Washington DC to advocate for Duchenne directly with Members of Congress. Our community’s presence in Washington has been critical in helping to move the…

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January 31, 2019

PPMD Kicks Off 2019 End Duchenne Tour in Las Vegas

PPMD is excited to kick off our 2019 End Duchenne Tour in Las Vegas this weekend! Combining each of the pillars that make up PPMD’s mission, the End Duchenne Tour brings updates on research, advocacy,…

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January 29, 2019

PPMD Designates Children’s Hospital of The King’s Daughters a Certified Duchenne Care Center

Today Parent Project Muscular Dystrophy (PPMD) named Children’s Hospital of The King’s Daughters (CHKD) the 23rd Certified Duchenne Care Center. PPMD’s Certified Duchenne Care Center Program, which supports standardized, comprehensive care and services for all people living…

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January 25, 2019

WATCH: Translarna® (ataluren) Development Update – Webinar Recording

On January 16, 2019, PTC Therapeutics joined Parent Project Muscular Dystrophy for a webinar update to provide the community with an overview of the Translarna development program with a focus on clinical trials that are…

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January 24, 2019

PPMD Awards $105,000 Grant to Explore A Mini-Dystrophin Gene Aimed at Protecting Cardiac Function

PPMD is excited to award the University of Missouri School of Medicine a grant for $105,000 to continue development of a minimized dystrophin gene aimed at protecting cardiac function in people with Duchenne. The project is led by Dongsheng…

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January 22, 2019

Capricor Announces Positive Outcomes from Comprehensive Multidisciplinary Meeting with FDA

Capricor Therapeutics announced that it had positive outcomes in its comprehensive multidisciplinary meeting with the FDA regarding development of the company’s novel cell therapy candidate, CAP-1002, after being placed on voluntary hold last month. To…

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