September 2, 2020

Santhera Exercises Option to Obtain Worldwide Rights to Vamorolone in Duchenne and All Other Indications

We congratulate Santhera as they have exercised the option to obtain worldwide rights to vamorolone in Duchenne and all other indications. The license gives Santhera worldwide rights to vamorolone which means Santhera is responsible for…

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August 25, 2020

Sarepta Announces FDA Acceptance of Casimersen NDA for Patients with Duchenne Amenable to Skipping Exon 45

PPMD is excited to learn that the FDA has accepted Sarepta’s New Drug Application (NDA) seeking accelerated approval for casimersen (SRP-4045) and provided a regulatory action date of February 25, 2021. Casimersen is a potential…

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August 24, 2020

Learnings from PPMD’s COVID-19 Pandemic Survey

In spring 2020, Parent Project Muscular Dystrophy (PPMD) released a survey to better understand the patient experience with care, trials, and access to approved therapies during the COVID-19 pandemic. Thank you to everyone who contributed…

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August 20, 2020

Sarepta Therapeutics Shares Summer 2020 Newsletter

Sarepta has shared the Summer 2020 issue of their newsletter, including updates on current Sarepta programs. Click here to download.

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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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