March 12, 2020

Update on Coronavirus Recommendations & PPMD Events

March 23, 2020 Update: The content below is a previous update from March 12, 2020. Please click here to view our most recent COVID-19 information & FAQs. PREVIOUS INFORMATION FROM MARCH 12, 2020: Update on…

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March 12, 2020

Solid Biosciences Provides Letter to the Duchenne Community: Data and IGNITE DMD Clinical Trial Update

Solid Biosciences Inc. provided an update on the IGNITE DMD phase I/II microdystrophin gene therapy clinical program. Solid shares that biomarker data from all three patients dosed in the 2E14 vg/kg cohort of IGNITE DMD…

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March 10, 2020

PPMD Leads Duchenne Community to Nation’s Capital for Annual Advocacy Conference

Parent Project Muscular Dystrophy (PPMD) heads to Washington, D.C., March 8–10, for our annual Advocacy Conference. Over 100 families are expected to join PPMD on Capitol Hill to help inform Members of Congress about the critical…

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March 9, 2020

Action Alert! Urge Congress to Support FY2021 Duchenne Appropriations

It’s that time of year again. Members of the House and Senate are working, right now, to develop and submit their policy priorities for the annual spending bills that will fund the NIH, CDC, FDA,…

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March 9, 2020

PPMD Hosts Leaders from NIH and DOD to Discuss Duchenne Community’s Work with Federal Agencies

With PPMD’s 2020 Advocacy Conference well underway, attendees participated in a special breakfast session earlier today reflecting on the progress our community has made since the first National Institutes of Health (NIH) workshop on Duchenne…

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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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