PPMD Benefit/Risk Study Results Published in Clinical Therapeutics - PPMD Community

PPMD Benefit/Risk Study Results Published in Clinical Therapeutics - PPMD Community: "PPMD is thrilled that results from the recent benefit/risk project our team led have been published in Clinical Therapeutics. The article concludes that caregivers are willing to accept a serious risk when balanced with noncurative slowing or stopping of the progression of muscle weakness, even absent improvement in lifespan. These preferences should inform the Food and Drug Administration’s (FDA’s) benefit–risk assessment of emerging Duchenne therapies."



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Lend Your Voice to the FDA Draft Guidance on Duchenne to Speed the Development of Therapies - PPMD Community

Lend Your Voice to the FDA Draft Guidance on Duchenne to Speed the Development of Therapies - PPMD Community: "For the past four months, over 80 experts in Duchenne (including patient representatives and groups, industry, clinicians, and researchers) have been working hard to produce draft guidance on Duchenne that we believe will ultimately speed the development of therapies. "



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Prosensa Announces A Regulatory Path Forward for Drisapersen as a Potential Treatment for Duchenne - PPMD Community

Prosensa Announces A Regulatory Path Forward for Drisapersen as a Potential Treatment for Duchenne - PPMD Community: "Prosensa Plans to Submit a New Drug Application to the FDA this Year; Dialogue with EMA continues with intent to seek approval"



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Congressional Committee That Funds the FDA Recognizes PPMD-Led Efforts on Benefit/Risk, Draft Guidance; Applauds Collaboration with Agency - PPMD Community

Congressional Committee That Funds the FDA Recognizes PPMD-Led Efforts on Benefit/Risk, Draft Guidance; Applauds Collaboration with Agency - PPMD Community: "For the first time ever, the appropriations bill that funds the Food and Drug Administration includes report language focused on Duchenne muscular dystrophy and championed by PPMD advocates."



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Webinar: Ataluren (Translarna) Update with PTC - PPMD Community

Webinar: Ataluren (Translarna) Update with PTC - PPMD Community: "In a webinar hosted by United Parent Project Muscular Dystrophy, Robert Spiegel, MD, FACP, chief medical officer of PTC Therapeutics, will present an overview of the positive opinion by the Committee for Medicinal Products for Human Use of the European Medicines Agency regarding the company’s application for a conditional marketing authorization of TranslarnaTM (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy, and what this decision means for families living in and outside the EU."



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» Prosensa update on regulatory discussions and ongoing plans for re-dosing Action Duchenne

» Prosensa update on regulatory discussions and ongoing plans for re-dosing Action Duchenne: "Prosensa today provided an update to the patient-led organisations. They have been encouraged in their discussions with the regulatory authorities and are on-track for a pathway forward by the end of June.

The team also informed us that they are also on-track for re-dosing in the first group of boys in the third quarter of this year. Re-dosing is taking a staged approach, under new or existing treatment protocols or via expanded access programs. In a first wave, they will initiate re-dosing at sites in both US and Europe and as stated previously are actively working with these sites and investigators to execute these plans."



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EMA's CHMP gives positive opinion for PTC Therapeutics' Translarna

EMA's CHMP gives positive opinion for PTC Therapeutics' Translarna: "PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that following its request for re-examination, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion regarding the company's application for a conditional marketing authorization of TranslarnaTM (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) in ambulatory patients aged five years and older."



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