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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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 13, 2020

Update from the 3rd Annual Duchenne Healthcare Professionals’ Summit

Moving Forward Together was the theme of PPMD’s 2020 Duchenne Healthcare Professionals’ Summit held late last month. It was an appropriate theme considering the audience of healthcare professionals in attendance who have consistently demonstrated their…

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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

Register Today for PPMD’s 2020 Advocacy Conference!

Registration for the PPMD Advocacy Conference is live! Join us March 8-10, 2020 in Washington, DC to advocate for Duchenne directly with your Members of Congress. Because of our community’s presence in Washington for the…

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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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