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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 […]

Researchers Isolate ST2 Protein as Potential Biomarker for Heart Disease in Duchenne Muscular Dystrophy

Researchers have found Duchenne muscular dystrophy (DMD) patients with heart disease have very high levels of ST2, an interleukin 1 receptor-like 1 protein. That makes this molecule a potential biomarker for cardiac disease in such patients. The study by scientists at Children’s National Health System in Washington, D.C., “Interleukin 1 Receptor-Like 1 Protein (ST2) is a Potential Biomarker […]

New Gene Therapy to Fix Dystrophin Deficiency in DMD Shows Promise in Mice, Study Shows

Researchers at the University of Missouri have developed a new method to efficiently deliver the correct form of dystrophin gene to muscles as a way to correct the faulty gene that characterizes Duchenne muscular dystrophy (DMD), a mouse study shows. Their study, “A Five-Repeat Micro-Dystrophin Gene Ameliorated Dystrophic Phenotype in the Severe DBA/2J-mdx Model of Duchenne Muscular Dystrophy,” appeared in […]

Microdystrophin Gene Therapy Shows Promise in Dogs with Duchenne Muscular Dystrophy, Study Shows

Injecting a smaller but functional form of the dystrophin gene, called microdystrophin, into dogs naturally affected by Duchenne muscular dystrophy allowed them to recover muscle strength and stabilized their overall disease symptoms, a new study shows. “This preclinical study demonstrates the safety and efficacy of microdystrophin, and makes it possible to consider developing a clinical trial in patients,” Caroline Le […]

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