January 21, 2020

Santhera Shares Winter 2020 Community Update

Santhera Pharmaceuticals shared a letter with the community, emphasizing that their main focus in the U.S. right now is to bring the SIDEROS clinical trial to full enrollment. Learn more about this clinical trial, including…

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January 15, 2020

WATCH: Update on Investigational Suvodirsen in Duchenne Muscular Dystrophy – Webinar Recording

On December 20, 2019, Parent Project Muscular Dystrophy (PPMD) and World Duchenne Organization (WDO) hosted an informational webinar with the Wave Life Sciences team to discuss their decision to discontinue development of suvodirsen for patients…

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January 15, 2020

PPMD’s Duchenne Drug Development Roundtable Creates Clinical Trial Harmonization Guidances

PPMD is excited to share the recently developed Clinical Trial Harmonization Guidances. These guidances are the result of PPMD’s Duchenne Drug Development Roundtable working collaboratively in the pre-competitive space to encourage better consistency across companies…

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January 8, 2020

Italfarmaco Shares Winter 2020 Newsletter

Italfarmaco has shared the Winter 2020 issue of their newsletter, including updates on the Givinostat Development Program. Click here to download.

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January 8, 2020

Catabasis Shares January 2020 Newsletter

Catabasis shares the latest edition of their community newsletter, including information about the Phase 3 PolarisDMD trial enrolling the expected patient population and a partnership with our friends at Duchenne UK to evaluate edasalonexent in…

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December 23, 2019

Sarepta Announces Partnership with Roche Outside the U.S. for its Investigational Micro-dystrophin Gene Therapy

Sarepta Therapeutics, Inc. today announced that Sarepta and Roche have entered into a licensing agreement providing Roche exclusive commercial rights to SRP-9001 (AAVrh74.MHCK7.micro-dystrophin), Sarepta’s investigational gene therapy for Duchenne muscular dystrophy, outside the United States….

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December 20, 2019

Sarepta Therapeutics Shares Winter 2019 Newsletter

Sarepta has shared the Winter 2019 issue of their newsletter, including updates on current Sarepta programs. Click here to download.

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December 20, 2019

WATCH: Better Todays. Stronger Tomorrows. – Webinar Recording

On December 19, 2019, PPMD had the privilege of hosting a conversation with Duchenne experts on the importance of optimal care in Duchenne, how care impacts every aspect of our mission to end Duchenne and our…

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December 19, 2019

Mallinckrodt Terminates BRAVE Study Investigating the Efficacy and Safety of MNK-1411 in Duchenne

PPMD is disappointed to learn that Mallinckrodt has made the decision to terminate their BRAVE Study (MNK14112096). Based on the company’s statement below, the decision appears to have been the result of low enrollment and…

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December 18, 2019

Solid Biosciences Provides Data Update from SGT-001 Development Program

Solid Biosciences Inc. provided an update on the IGNITE DMD phase I/II microdystrophin gene therapy clinical program. Solid shares that the status of the sixth patient dosed in the higher 2E/14 vg/kg, who experienced the…

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