February 11, 2019

PPMD Designates University of Rochester Medical Center as a Certified Duchenne Care Center

Today Parent Project Muscular Dystrophy (PPMD) named the University of Rochester Medical Center (URMC) the 24th Certified Duchenne Care Center. This expanding program of elite centers, created in 2014 as part of PPMD’s Certified Duchenne Care Center Program, reviews and…

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February 8, 2019

How Do I Find a Clinical Trial That’s Right for Me?

Are you getting confused by all the news and social media posts about clinical trials? Not sure which trials or studies may be appropriate for you or your child? You are not alone! Even healthcare…

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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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