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Research Updates

Sarepta Therapeutics and Nationwide Children’s Hospital Announce FDA Clearance of IND for Micro-Dystrophin Gene Therapy Program for the Treatment of Duchenne Muscular Dystrophy

CAMBRIDGE, Mass., Nov. 06, 2017 (GLOBE NEWSWIRE) — Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a commercial-stage biopharmaceutical company focused on the discovery and development of precision genetic medicines to treat rare neuromuscular diseases, today announced that Nationwide Children’s Hospital has received clearance from the U.S. Food and Drug Administration (FDA) for its Investigational New Drug (IND) application […]


A Letter from PTC Therapeutics

Dear Duchenne Community, As you might have seen, the Office of Drug Evaluation I of the FDA provided a Complete Response Letter (CRL) for the application to market ataluren in the United States. This means that the FDA believes ataluren cannot be approved at this time. We are extremely disappointed and we strongly disagree with […]


FDA Rejects New Drug Application for Translarna to Treat DMD

The U.S. Food and Drug Administration decided not to approve Translarna (ataluren), by PTC Therapeutics, as a treatment for specific types of Duchenne muscular dystrophy caused by “nonsense” mutations. Although not entirely unexpected, the decision was disappointing to many in the Duchenne community. On Sept. 28, an FDA advisory panel concluded that evidence to support Translarna’s effectiveness as a therapy […]


Immune Cells Soak Up Duchenne MD Therapy Exondys 51, Prolonging Its Work, Study Reports

The newly approved Duchenne muscular dystrophy therapy Exondys 51 (eteplirsen) stays in immune cells several days after leaving the blood, continuing to help regenerate muscle fibers, according to a study in mice. Researchers at the Children’s National Health System in the Washington area said the findings on immune cells known as macrophages shed light on the cell mechanisms that […]


Advanced Gene-Editing Technology CRISPR-Gold Repairs DMD Mutation in Mice

A new version of the CRISPR-Cas9 gene-editing technology called CRISPR-Gold has successfully restored the correct sequence of the dystrophin gene in a mouse model of Duchenne muscular dystrophy (DMD), a new study revealed. Researchers found that an injection of CRISPR-Gold into DMD mice led to an 18-times-higher correction rate and a two-fold increase in a strength […]


Intravenous Delivery of CAP-1002 Shown Effective Ahead of Upcoming HOPE 2 Trial for DMD

The biotechnology company Capricor Therapeutics recently presented data that support the use of its peripheral intravenous delivery method in the upcoming HOPE-2 clinical trial testing the investigational agent CAP-1002 as a potential therapy for Duchenne muscular dystrophy (DMD). The data were presented during a poster session at the Alliance for Regenerative Medicine’s Cell and Gene Meeting on the […]


Capricor Therapeutics Presents Positive Six-Month Results in Duchenne Muscular Dystrophy at World Muscle Society International Congress

SAINT MALO, France, Oct. 4, 2017 /PRNewswire/ — Capricor Therapeutics, Inc. (NASDAQ: CAPR), in its presentation today at the 22nd Annual International Congress of the World Muscle Society, reported that teens and young men in the advanced stages of Duchenne muscular dystrophy (DMD) experienced meaningful improvements in cardiac and upper limb function after a single dose of Capricor’s lead investigational […]


Sarepta on ‘Mission’ It Intends to Accomplish and Soon, CEO Doug Ingram Says in Interview

With one exon-skipping therapy — Exondys 51 (eteplirsen) — approved for Duchenne muscular dystrophy (DMD) and others in clinical trials, it’s easy to envision Sarepta Therapeutics as the “exon-skipping company.” But the idea of current exon-skipping treatments as the ultimate for DMD couldn’t be further from what Sarepta says it wants to achieve. In fact, […]


Jerry Mendell, in PPMD Webinar, Details Plans for Possible Trial of Duchenne Gene Therapy

In a recent webinar, Dr. Jerry Mendell with Nationwide Children’s Hospital rolled out plans for a Phase 1/2a clinical trial of a new gene therapy for Duchenne’s muscular dystrophy (DMD). Final go-ahead for the trial awaits U.S. Food and Drug Administration (FDA) approval of an Investigational New Drug (IND) application1 made for the therapy and its testing. But Mendell is […]


Phrixus Pharmaceuticals announces the first patient with Duchenne muscular dystrophy (DMD) to meet the 15-month mark on Carmeseal-MD™ (Poloxamer 188 NF)

First patient to be treated with Carmeseal-MD (P-188 NF) outside of the United States through Phrixus’s Expanded Access Program with Ethicor Pharma Ltd. – ANN ARBOR, MI – X September 2017 – Phrixus Pharmaceuticals, Inc. (“Phrixus”), a company focused on therapies for Duchenne muscular dystrophy and heart failure, today announced that the Oirst patient with […]