May 3, 2021

Sarepta Therapeutics Reports Positive Clinical Results from Phase 2 MOMENTUM Study of SRP-5051

Sarepta has announced positive results from Part A of the MOMENTUM study (Study 5051-201), a Phase 2 clinical trial of SRP-5051, its next-generation treatment for individuals with Duchenne who are amenable to exon 51 skipping. Part…

Learn More

February 25, 2021

FDA Grants Accelerated Approval to AMONDYS 45™ (Casimersen)

PPMD is excited to learn that the FDA has granted accelerated approval to AMONDYS 45™ (casimersen), a treatment of Duchenne in patients who have a confirmed mutation of the DMD gene that is amenable to…

Learn More

December 7, 2020

Sarepta Announces Positive Clinical Results from MOMENTUM Phase 2 Clinical Trial of SRP-5051

Sarepta has announced positive results from the ongoing MOMENTUM study of SRP-5051, its next-generation treatment for individuals with Duchenne who are amenable to exon 51 skipping. This is the first clinical data from SRP-5051, an…

Learn More

October 8, 2020

Sarepta Therapeutics Shares Fall 2020 Newsletter

Sarepta has shared the Fall 2020 issue of their newsletter, providing updates on community programs and highlighting their new class of Route 79 scholarship recipients. Click here to download.

Learn More

September 2, 2020

WATCH: NS Pharma Announcement of VILTEPSO (viltolarsen) – Webinar Recording

NS Pharma joined PPMD for a webinar on August 26, 2020 to provide an update about VILTEPSO™ from Ben Yungher, PhD, NS Pharma’s Medical Science Liaison, and its associated patient support program from Randy McGonigal,…

Learn More

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…

Learn More

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.

Learn More

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…

Learn More

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…

Learn More

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…

Learn More