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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March 6, 2020

Important Update: PPMD’s End Duchenne Tour in Atlanta, GA on March 14

With the coronavirus (COVID-19) top of mind in headlines across the country, in addition to the continued risk for flu and other respiratory illnesses, PPMD has consulted with clinicians from many of our Certified Duchenne…

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March 6, 2020

Event Spotlight: Kick to Cure Duchenne

Looking to do an easy and fun event in your community? Look no further! We’ve got the event for you. Host a community kickball tournament! The Labbadia family hosted their annual Kick to Cure Duchenne…

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March 6, 2020

PPMD Applauds Reintroduction of Bipartisan Legislation to Enhance Role of the Patient in Drug Development

Parent Project Muscular Dystrophy (PPMD) applauds this week’s reintroduction of legislation called the BENEFIT Act, bipartisan legislation that will strengthen current law requiring the Food and Drug Administration consider the voice of patients and caregivers…

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March 5, 2020

Update on PPMD’s 2020 Advocacy Conference

As many of you know, PPMD and a number of families from the Duchenne community plan to descend upon our Nation’s Capital this weekend to help make sure that Duchenne research is included in the…

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March 4, 2020

WATCH: Connect with the Experts: Cardiac Care & Research – Webinar Recording

Last week, PPMD hosted the first quarterly Connect with the Experts webinar around cardiac care and research. Dr. Linda Cripe (Nationwide Children’s), Dr. Lee Sweeney (University of Florida), and PPMD’s Eric Camino (Director of Research…

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March 3, 2020

PPMD’s Pat Furlong Presents at FNIH Biomarkers Consortium

Last month, PPMD’s Founding President & CEO Pat Furlong presented at the Foundation for the National Institutes of Health (FNIH) Biomarkers Consortium, addressing the use of digital health technologies to improve therapeutic research and development….

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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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