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 19, 2020

Check out the schedule for PPMD’s Virtual Conference!

There’s still time to register for PPMD’s Virtual Conference! Though we can’t connect in person this year, there was no way PPMD could let the summer go without hosting our Annual Conference. So this week,…

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July 16, 2020

Celebrating PPMD’s “Change It Champion” Award-Winner Claudia Senesac, PT, PhD, PCS

Celebrating PPMD’s “Change It Champion” Award-Winner Claudia Senesac, PT, PhD, PCS PPMD is excited to celebrate our recent Change It Champion Award-winner Claudia Senesac, PT, PhD, PCS, from University of Florida, in recognition of her…

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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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July 14, 2020

WATCH: Family Friday – Creating a Unique Virtual Conference Experience

Family Friday: Creating a Unique Virtual Conference Experience With PPMD’s Annual Conference going virtual this year, we wanted to explore ways each family could tailor their conference experience to suit their needs and interests. During…

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July 13, 2020

PPMD’s 2020 Virtual Conference – Our Hub is Live!

PPMD’s 2020 Virtual Conference – Our Hub is Live! If you haven’t registered for PPMD’s 2020 Virtual Annual Conference — now’s the time to do so! Our Conference Hub is now open! This is your opportunity to create…

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July 7, 2020

PPMD Signs Onto Telehealth Letter to Congress

PPMD is proud to have joined 340 organizations in signing a letter urging Congressional leaders to make telehealth flexibilities created during the COVID-19 pandemic, permanent. The letter sent a powerful message to Congress outlining the…

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