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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July 7, 2020

NS Pharma Announces Launch of Patient Support Hub

NS Pharma, Inc. announced the launch of NS Support for patients with Duchenne and their healthcare providers. NS Support will provide a resource for patients and doctors to get more information once NS Pharma has…

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July 2, 2020

Sarepta Therapeutics Signs Agreement with Hansa Biopharma for Imlifidase

PPMD is encouraged to learn that Sarepta Therapeutics announced an agreement with Hansa Biopharma, a company that has developed an IgG antibody-cleaving enzyme called imlifidase. Imlifidase is an investigational product initially developed to aid in…

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June 26, 2020

Sarepta Completes Submission of New Drug Application Seeking Approval of Casimersen (SRP-4045)

PPMD is excited to learn that Sarepta Therapeutics has completed the submission of a rolling New Drug Application (NDA) to the FDA seeking accelerated approval for casimersen (SRP-4045). Casimersen, a phosphorodiamidate morpholino oligomer (PMO), is…

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