June 2, 2022

NS Pharma Announces Publication of Long-Term Efficacy and Safety Data of VILTEPSO® (Viltolarsen) in Duchenne

NS Pharma, Inc. has announced the publication of long-term efficacy and safety data from the open-label extension trial of a Phase 2 study of VILTEPSO® (viltolarsen) in participants with Duchenne. We are pleased to see…

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May 17, 2022

Nationwide Children’s Announces Restoration of Full-Length Dystrophin Using Dup 2 Gene Therapy Approach

PPMD is very excited to see promising results shared from Nationwide Children’s scAAV9.U7.ACCA gene therapy trial, a Phase 1/2 study aiming to induce exon skipping for individuals with a duplication of exon 2 to restore full-length…

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May 13, 2022

Entrada Therapeutics Shares New Data on ENTR-601-44 Exon Skipping Therapy

Entrada Therapeutics has shared new data from a preclinical study evaluating ENTR-601-44, an exon-skipping therapy set to target individuals with Duchenne amenable to exon 44 skipping. We are pleased to hear new data on this…

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April 22, 2022

Astellas Gene Therapies Announces plans to discontinue development of Duchenne gene therapy programs

Last night, based on recent preclinical data, Astellas Gene Therapies announced that the research and development of gene therapy programs AT702, AT751 and AT753 for Duchenne Muscular Dystrophy have been terminated. • AT702 was designed…

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January 20, 2022

Dyne Therapeutics Announces FDA Clinical Hold on IND Application for DYNE-251 in Duchenne

Dyne Therapeutics recently shared that the FDA issued a clinical hold on their Investigational New Drug (IND) application for the Phase I/II study of their exon skipping therapy utilizing a conjugated monoclonal antibody DYNE-251. For…

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September 29, 2021

Wave Life Sciences Announces Initiation of Dosing in Phase 1b/2a Duchenne Clinical Trial of WVE-N531 Starting in Canada and the UK

  This morning, Wave Life Sciences Ltd. announced the initiation of dosing in a Phase 1b/2a clinical trial expecting to enroll 15 boys, evaluating investigational WVE-N531 as a treatment for boys with Duchenne muscular dystrophy…

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August 12, 2021

NS Pharma Shares Community Letter

August 12, 2020, marked the approval of Viltepso, the 4th FDA approved therapy for Duchenne. NS Pharma has posted the following community letter marking the occasion. Today we recognize the one-year anniversary of this approval….

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May 12, 2021

NS Pharma Announces VILTEPSO® Long-Term Trial Data to be Presented at PPMD’s 2021 Virtual Annual Conference

NS Pharma has announced that they will be presenting new, long-term efficacy and safety data from the open-label extension of a Phase 2 study of VILTEPSO® (viltolarsen) at PPMD’s 2021 Virtual Annual Conference next month….

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May 3, 2021

WATCH: Viltepso – A Treatment Option for Patients Amenable to Exon 53 Skipping (Webinar Recording)

NS Pharma joined PPMD for a webinar on April 22, 2021 to provide an update about VILTEPSO™, a treatment of Duchenne in patients who have a confirmed mutation of the DMD gene that is amenable to…

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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…

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