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HOSPITAL AND RESEARCHERS AWARDED $2.2M FOR DUCHENNE GENE THERAPY INVESTIGATION

September 9, 2017

The nonprofit Duchenne muscular dystrophy (DMD) research and advocacy organization Parent Project Muscular Dystrophy (PPMD) awarded a $2.2 million grant to Jerry Mendell, MD, PhD; co-principal investigator Louise Rodino-Klapac, PhD; and Nationwide Children’s Hospital in Columbus, Ohio, where they both work.
The funding will support Mendell, Rodino-Klapac, and their research team’s investigation into gene therapy as a potential Duchenne MD treatment.
Mendell is a professor of pediatrics and neurology and director of the center for gene therapy at Nationwide Children’s Hospital’s Research Institute, and Rodino-Klapac heads the Rodino-Klapac Gene Therapy Lab for Muscular Dystrophies, which is focused on developing gene therapy-based vectors for the treatment of various neuromuscular disorders. She is also an assistant professor at the The Ohio State University School of Medicine.

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CHMP RECOMMENDS RENEWAL OF TRANSLARNA’S™ MARKETING AUTHORIZATION FOR NONSENSE MUTATION DUCHENNE MUSCULAR DYSTROPHY BASED ON CONTINUED POSITIVE BENEFIT-RISK ASSESSMENT

November 11, 2016

PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended the renewal of the conditional marketing authorization of Translarna™ (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) in ambulatory patients five years and older. In connection with the renewal, the marketing authorization will include a specific obligation to conduct an additional long-term post-authorization trial.

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PTC THERAPEUTICS ANNOUNCES NEW ANALYSES THAT SUPPORT THE POTENTIAL BENEFIT OF ATALUREN IN PRESERVING LUNG FUNCTION IN NON-AMBULATORY NONSENSE MUTATION DUCHENNE MUSCULAR DYSTROPHY PATIENTS

October 6, 2016

PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced new data supporting the potential benefit of ataluren in preserving lung function in non-ambulatory nonsense mutation Duchenne muscular dystrophy patients (nmDMD). The results, which are being presented today as part of a company-sponsored symposium, are based on PTC’s analyses of lung function data from one of PTC’s ongoing open-label extension studies (Study 019) versus natural history data from a comparable non-ambulatory cohort.

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SAREPTA THERAPEUTICS ANNOUNCES FIRST PATIENT DOSED IN PHASE III CLINICAL TRIAL OF SRP-4045 AND SRP-4053 FOR THE TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY AMENABLE TO EXON 45 OR 53 SKIPPING

September 28, 2016

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a developer of innovative RNA-targeted therapeutics, today announced the first patient dosed in the phase III clinical trial of SRP-4045 and SRP-4053 in patients with Duchenne muscular dystrophy amenable to exon 45 or 53 skipping.

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SAREPTA THERAPEUTICS ANNOUNCES FDA ACCELERATED APPROVAL OF EXONDYS 51™ (ETEPLIRSEN) INJECTION, AN EXON SKIPPING THERAPY TO TREAT DUCHENNE MUSCULAR DYSTROPHY (DMD) PATIENTS AMENABLE TO SKIPPING EXON 51

September 19, 2016

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a developer of innovative RNA-targeted therapeutics, today announced that the U.S. Food and Drug Administration (FDA) has granted accelerated approval for EXONDYS 51™ (eteplirsen) as a once weekly intravenous infusion of 30 milligrams per kilogram for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation in the DMD gene that is amenable to exon 51 skipping.

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SAREPTA THERAPEUTICS ANNOUNCES FDA ADVISORY COMMITTEE MEETING TO REVIEW ETEPLIRSEN AS A TREATMENT FOR DUCHENNE MUSCULAR DYSTROPHY AMENABLE TO EXON 51 SKIPPING

December 18, 2015

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a developer of innovative RNA-targeted therapeutics, today announced that the Peripheral and Central Nervous System Drugs Advisory Committee of the U.S. Food and Drug Administration (FDA) will review Sarepta’s New Drug Application (NDA) for eteplirsen, on January 22, 2016. The Prescription Drug User Fee Act (PDUFA) action date for completion of FDA review of the eteplirsen NDA is February 26, 2016.

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BIOMARIN ANNOUNCES THAT FDA HAS ADVISED IT WILL NOT TAKE ACTION ON THE KYNDRISA™ (DRISAPERSEN) NEW DRUG APPLICATION BY THE PDUFA DATE

December 18, 2015

BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) announced today that the U.S. Food and Drug Administration (FDA) has notified the Company that they had not yet completed their review process and would be unable to take an action by the Prescription Drug User Fee Act (PDUFA) action date for KyndrisaTM (drisapersen) of December 27, 2015, and anticipate taking action in early January 2016.

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SPELL CHECKING NATURE: VERSATILITY OF CRISPR/CAS9 FOR DEVELOPING TREATMENTS FOR INHERITED DISORDERS

December 10, 2015

Clustered regularly interspaced short palindromic repeat (CRISPR) has arisen as a frontrunner for efficient genome engineering. How- ever, the potentially broad therapeutic implications are largely unexplored.

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FDA ADVISORY COMMITTEE DISCUSSES CLINICAL DATA PACKAGE FOR BIOMARIN’S KYNDRISA(TM) (DRISAPERSEN) FOR THE TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY AMENABLE TO EXON 51 SKIPPING

November 24, 2015

BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) announced today that the Peripheral and Central Nervous System Drugs Advisory Committee of the U.S. Food and Drug Administration (FDA) met to discuss the data submitted to support the New Drug Application (NDA) for KyndrisaTM (drisapersen) for the treatment of Duchenne muscular dystrophy (Duchenne) amenable to exon 51 skipping.

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