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 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 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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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 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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June 15, 2020

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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May 11, 2020

Sarepta and Dyno Therapeutics Announce Agreement to Develop Next-Generation Gene Therapy Vectors

Sarepta Therapeutics and Dyno Therapeutics announced an agreement to develop next-generation gene therapy vectors for muscle diseases. PPMD is pleased to see Sarepta’s continued investment and contribution in enhancing the future of gene therapy technologies…

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April 13, 2020

Sarepta Therapeutics Shares COVID-19 Resources

Sarepta Therapeutics has shared a new COVID-19 resource page on its website, along with a new video providing contact phone numbers for those whom may have questions about continuity of access to Sarepta sponsored clinical…

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March 19, 2020

Sarepta Therapeutics Shares Community Update on COVID-19

Sarepta Therapeutics has provided the community with an update addressing questions from the community regarding COVID-19, including updates on current clinical trials and accessing approved therapies. Read Sarepta’s Update March 19, 2020 Dear Duchenne and…

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