News

July 7, 2017July 7, 2017

Experts Review Current and Potential Treatments for Duchenne Muscular Dystrophy

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Experts provided an overview of the many approaches to counteract the loss of muscle mass, inflammation, fibrosis, calcium overload, oxidative stress, and ischemia (inadequate blood supply) in Duchenne muscular ... Read more

July 6, 2017July 6, 2017

Heart Failure Diagnosis and Therapy Reviewed in Duchenne Muscular Dystrophy Patients

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End-stage heart failure is increasingly becoming the main cause of death in patients with Duchenne muscular dystrophy (DMD). Because cardiomyopathy, an abnormal heart condition, is difficult to detect, a ... Read more

July 5, 2017

Linker Proteins Restore Muscle Cell Function in Congenital MD Animal Models, Study Shows

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Scientists have designed two proteins that stabilized muscle cell membrane support and maintained the structure of muscle fibers in animal models of congenital muscular dystrophy (MD). The finding, from ... Read more

June 30, 2017June 30, 2017

6-Month Results of Study into Stem Cell Therapy for Heart Disease in Duchenne Patients Part of PPMD Conference

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Interim results from the Phase 1/2 trial clinical trial investigating CAP-1002 as a treatment for heart disease associated with Duchenne muscular dystrophy (DMD) are being presented today at the 2017 Parent Project Muscular ... Read more

June 29, 2017June 29, 2017

Parent Project Muscular Dystrophy Gathers Inflammation, Immunity Experts for Duchenne Workshop in Chicago

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Parent Project Muscular Dystrophy (PPMD) has added a pre-conference workshop to this year’s 23rd Annual Connect Conference, inviting international medical experts to discuss inflammation and immunity in Duchenne muscular dystrophy (DMD). ... Read more

June 28, 2017

Earlier Start of Corticosteroids in DMD Linked to Earlier Heart Disease Onset, Study Suggests

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Researchers say it is important to carefully assess the timing of the start of corticosteroid treatment for Duchenne muscular dystrophy (DMD) in children and its potential association with disease outcomes. ... Read more

June 27, 2017

Pharmaceutical Partners Invest $6M in Eloxx to Advance Therapy for Duchenne MD

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Eloxx Pharmaceuticals received a $6 million investment from industry partners to advance a novel therapy for genetic diseases caused by nonsense mutations, including Duchenne muscular dystrophy (DMD), raising the ... Read more

June 26, 2017June 26, 2017

Edasalonexent Could Reduce Functional Decline in Boys with DMD, MoveDMD Phase 2 Results Suggest

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Results of the Phase 2 MoveDMD trial showed that Catabasis Pharmaceuticals’ candidate drug edasalonexent (CAT-1004) can reduce the rate of functional decline in boys ages 4–7 with Duchenne muscular dystrophy (DMD). The recent ... Read more

June 23, 2017

UK Agency Grants Raxone Temporary Approval to Treat Certain DMD Patients

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Raxone (idebenone) has been granted temporary scientific approval to be used to treat certain patients with Duchenne muscular dystrophy (DMD) in the United Kingdom (UK). The UK’s Medicines and ... Read more

June 22, 2017

Sarepta and Genethon Will Collaborate to Develop DMD Gene Therapy

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Sarepta Therpeutics and Genethon have signed a research collaboration agreement to jointly develop a gene therapy for Duchenne muscular dystrophy (DMD). Created by the AFM-Telethon, the French Muscular Dystrophy ... Read more