August 19, 2020

PPMD & Duchenne Parent Project NL Support Duchenne-Specific Submissions to Journal of Neuromuscular Disease

PPMD is proud to join our friends at Duchenne Parent Project NL in supporting the Duchenne-specific submissions to the Journal of Neuromuscular Diseases exploring preclinical and clinical studies that might not have met expectation. Of…

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August 13, 2020

2020 #PPMDConference Presentations

2020 PPMD Annual Conference Presentations JULY 22 – JULY 25, 2020 | Virtual Conference PPMD’s first Virtual Annual Conference was incredible with nearly 2500 families, care providers, researchers, scientists, and industry partners joining us for…

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August 11, 2020

FibroGen Announces First Patient Enrolled in Pamrevlumab Phase 3 Clinical Trial

FibroGen, Inc. announced the initiation of LELANTOS, a Phase 3, randomized, double-blind, placebo-controlled trial of pamrevlumab or placebo in combination with systemic corticosteroids in patients with non-ambulatory Duchenne. PPMD is excited to see FibroGen’s continued…

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August 8, 2020

FDA Grants Accelerated Approval to Viltepso (viltolarsen)

PPMD is thrilled to learn that the FDA has granted accelerated approval to NS Pharma’s VILTEPSO™ (viltolarsen), a treatment of Duchenne in patients who have a confirmed mutation of the DMD gene that is amenable…

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August 6, 2020

Have You Participated in a Clinical Trial That Was Terminated?

In an effort to learn more about the patient and family experience around clinical trials that have stopped or were terminated, PPMD and Duchenne UK have collaborated on a survey to learn more about your…

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July 24, 2020

Solid Biosciences Provides Update Regarding the IGNITE DMD Phase I/II Clinical Hold

Tonight at PPMD’s Virtual Annual Conference, Solid Biosciences shared that their IGNITE DMD Phase I/II clinical trial will remain on clinical hold. They also issued a press release here. During this hold, Solid will continue to…

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July 24, 2020

Catabasis Shares July 2020 Newsletter

Catabasis Pharmaceuticals has shared the latest edition of their community newsletter, which highlights the value of the North Star Ambulatory Assessment in Duchenne. Click here to download the PDF.

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July 24, 2020

Vita Therapeutics receives Orphan Drug Designation from FDA

Vita Therapeutics, Inc. recently announced that it received orphan drug designation from the FDA for VTA-110, a novel regenerative therapy for the treatment of Duchenne. VTA-110, is a potential first-in-class allogenic iPSC-based therapy that has…

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July 24, 2020

Sarepta Therapeutics Receives Fast Track Designation for SRP-9001 Micro-Dystrophin Gene Therapy

PPMD was excited to learn at yesterday’s Virtual Annual Conference that Sarepta Therapeutics was granted Fast Track designation for SRP-9001 by the FDA. SRP-9001 is an investigational gene transfer therapy intended to deliver its micro-dystrophin-encoding…

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July 14, 2020

Win a NEW Smartphone!

Win a NEW Smartphone! During PPMD’s Virtual Annual Conference in July, The Duchenne Registry will be giving away a brand new smartphone (iPhone or Android) to one lucky registrant! All you have to do is…

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