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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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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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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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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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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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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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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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WATCH: Antisense Therapeutics Phase II Trial Final Data Results – Webinar Recording

Antisense Therapeutics recently announced that their Phase II clinical trial of ANP’s immunomodulatory therapy, ATL1102 for Duchenne in non-ambulatory patients has met its primary endpoint, confirming the safety and tolerability of ATL1102 for advancement into…

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Sarepta Announces Positive Safety and Efficacy Data from SRP-9001 Gene Therapy Trial Published

Sarepta Therapeutics today announced safety and tolerability data at one year from four Duchenne clinical trial participants who received SRP-9001 micro-dystrophin (AAVrh74.MHCK7.micro-dystrophin) have been published in JAMA Neurology. PPMD is proud to have been an…

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WATCH: Capricor’s HOPE-2 Final 12-Month Data – Webinar Recording

Capricor Therapeutics recently announced positive final 12-month results from the HOPE-2 Trial. HOPE-2 was a randomized, double-blind, placebo-controlled study focused on later-stage Duchenne patients. On June 3, 2020, Capricor joined PPMD for a webinar to…

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