March 20, 2019

Register Today for PPMD’s 2019 Annual Conference!

PPMD’S 2019 ANNUAL CONFERENCE JUNE 26 – JUNE 30, 2019 ORLANDO, FL 2019 marks PPMD’s 25th Annual Conference. No other Duchenne conference comes close to the experience of the PPMD Annual Conference. Families, physicians, researchers, caregivers,…

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March 19, 2019

Cure SMA & PPMD Announce Strategic Collaboration with Prometheus Research

PPMD is thrilled to announce our strategic collaboration with Cure SMA, one of the world’s leading rare disease patient advocacy organizations. Like PPMD, Cure SMA is looking to advance data collection and analysis on their…

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March 13, 2019

Updates from PPMD’s 2019 Gene Therapy Policy Forum

During PPMD’s Annual Advocacy Conference we had the opportunity to host a Gene Therapy Forum with expert clinicians, researchers, industry stakeholders, and government representatives taking part in the discussion. This meeting continues PPMD’s in-depth look…

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March 11, 2019

Respiratory Management in Duchenne

Improved respiratory (breathing) management over the past 10 years has significantly improved the quality and length of life for people living with Duchenne. In October 2018, articles discussing specific areas of Duchenne care were released. One…

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March 5, 2019

Action Alert! Continue Progress in the Fight Against Duchenne

Thanks to the work of our Duchenne community over the past two decades, Congress has regularly supported important programs and policies to advance research, public health, drug development, and other Duchenne priorities. To ensure that we…

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February 28, 2019

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

Parent Project Muscular Dystrophy (PPMD) heads to Washington, D.C., March 3–5, for their annual Advocacy Conference. Over 100 families are expected to join PPMD on Capitol Hill to help inform Members of Congress about the…

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February 27, 2019

Roche/Genentech General Program Update Letter to the Duchenne Community

As Roche/Genentech continues to advance its Phase 2/3 clinical development program for RG6206, an adnectin fusion protein designed to inhibit myostatin and stimulate muscle growth in ambulatory people with Duchenne, the company has provided an…

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February 25, 2019

Rehabilitation, Therapy and Appeal Letters for Medical Equipment

Assistive devices and adaptive equipment are supposed to make life easier. So why is getting this equipment so hard? Every month, PPMD members get notes from parents and rehabilitation teams who are frustrated with the…

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February 25, 2019

Santhera’s SYROS Study Shows Long-term Efficacy with Idebenone in Slowing Respiratory Function Loss

Santhera Pharmaceuticals announced positive results from the SYROS study demonstrating that long-term treatment with idebenone consistently reduced the rate of respiratory function loss in patients with Duchenne for up to 6 years in a real-world…

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February 20, 2019

PPMD Contributes to Clinical Practice Recommendations for Managing Adrenal Insufficiency Risk in Duchenne

Long term glucocorticoid (steroid) therapy has many clear benefits such as improving muscle strength and maintaining cardiac and pulmonary function. However, prescribed steroid (prednisone, Emflaza, or deflazacort) regimens need to be followed closely to avoid…

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