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Research Updates

#AAN2018 – Early Data Supports Golodirsen in DMD Patients Amenable to Exon 53 Skipping

A potential next-generation therapy, golodirsen (SRP-4053) facilitates and increases dystrophin production in Duchenne muscular dystrophy (DMD) patients, an interim analysis of a Phase 1/2 trial being presented at the 2018 American Academy of Neurology (AAN) Annual Meeting in Los Angeles reports. These findings, in  “Golodirsen Induces Exon Skipping Leading to Sarcolemmal Dystrophin Expression in Duchenne Muscular Dystrophy Patients With Mutations […]

Summit Presents New 24-Week Analyses from PhaseOut DMD at the 2018 American Academy of Neurology Annual Meeting

OXFORD, United Kingdom and CAMBRIDGE, Mass., April 20, 2018 (GLOBE NEWSWIRE) — Summit Therapeutics plc (NASDAQ:SMMT) (AIM:SUMM) announces the presentation of new 24-week interim analyses from PhaseOut DMD, a Phase 2 open-label, multi-centre clinical trial of the utrophin modulator ezutromid in Duchenne muscular dystrophy (‘DMD’), at the 70th American Academy of Neurology Annual Meeting (‘AAN’). These new […]

#AAN2018 – Clinical Trials Re-Analysis Shows Emflaza Potential to Preserve Motor Function in Boys with DMD

Treatment with Emflaza (deflazacort) is better at preserving motor function in patients with Duchenne muscular dystrophy (DMD) than standard of care corticosteroid treatments, according to a re-analysis of data from two clinical trials. The new findings follow a reassessment of pooled data obtained in the placebo group of the Study 007 Phase 2b (NCT00592553) and ACT DMD Phase 3 (NCT01826487) […]

#AAN2018 — Exondys 51 Found to Be Safe in Young Infant with DMD, Preliminary Data Shows

Treatment with Exondys 51 (eteplirsen) was found to be safe and well-tolerated in a very young infant with Duchenne muscular dystrophy (DMD), preliminary data of a follow-up study shows. Initial results of the study will be the subject of a poster presentation at the 2018 Annual Meeting of the American Academy of Neurology (AAN), taking place April 21-27 in Los Angeles. The presentation […]

NIH Agency Pioneers Collaborative Research into Rare Diseases

A little-known government entity within the National Institutes of Health (NIH) is helping to lead U.S. efforts to speed up the development of therapies for some 7,000 rare diseases. The Office of Rare Diseases Research (ORDR), headquartered in Bethesda, Maryland, was established in 1993 within the NIH Office of the Director. In 2011, it became part of the NIH’s National […]

Pfizer launches Duchenne gene therapy study, joining Sarepta and Solid

Pfizer has officially entered the race to cure Duchenne muscular dystrophy with gene therapy, joining Cambridge-based Sarepta Therapeutics and Solid Biosciences as the only companies to use the experimental technology on a boy with the muscle-wasting disease. Pfizer (NYSE: PFE) announced on Thursday that the first patient in a small, early-stage trial of its gene therapy drug had […]

Duchenne Muscular Dystrophy: As Survival Increases, New Focus on Quality of Life

David Birnkrant, MD Professor of Pediatrics, Case Western Reserve University School of Medicine Director of Pediatric Pulmonology & Student Education, MetroHealth Medical Center What is the background for this study? What are the main findings? Response: This study updates guidance on all aspects of the multi-disciplinary care of patients with Duchenne muscular dystrophy (DMD). The […]

DMD Trial of Dystrophin Gene Therapy SGT-001 Put on Clinical Hold by FDA

The U.S. Food and Drug Administration has placed a clinical hold on a Phase 1/2 trial testing SGT-001, a dystrophin gene therapy intended to treat Duchenne muscular dystrophy (DMD), after the first patient dosed was hospitalized with a possibly serious reaction. Solid Biosciences, the therapy’s developer, announced that within days of being given SGT-001 at 5E13 vg/kg […]

Approval Received for Phase II Trial of ATL1102 in Duchenne Muscular Dystrophy

MELBOURNE, Australia, February 28, 2018 ( – Antisense Therapeutics (“ANP” or the “Company”) is pleased to advise that the Company has received approval from the Royal Children’s Hospital (RCH), Melbourne Human Research Ethics Committee, to undertake a Phase II clinical trial of its immunomodulatory therapy, ATL1102, in patients with Duchenne Muscular Dystrophy (DMD), an incurable muscle-wasting […]