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September 18, 2019September 18, 2019

Acceleron Stops Clinical Development of ACE-083 as Treatment for FSHD

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Due to a lack of efficacy, Acceleron Pharma is halting the clinical development of its ACE-083 candidate for the treatment of facioscapulohumeral muscular dystrophy (FSHD) . Topline results from a ... Read more

September 16, 2019September 16, 2019

New Blood Biomarkers of DMD and Glucocorticoid Response Identified

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Researchers have identified a new set of blood biomarkers of Duchenne muscular dystrophy (DMD) and of response to standard glucocorticoid therapy. The study, conducted by U.S. and Canadian researchers, ... Read more

September 13, 2019September 13, 2019

Microdystrophin Gene Therapy Improves with nNOS Domain, Review Study Says

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A review study highlights the benefits of adding an extra protein domain — called neuronal nitric oxide synthase (nNOS) — to microdystrophin gene therapy. nNOS is an important player ... Read more

September 11, 2019September 11, 2019

MDA Awards $6.6M to 25 Scientists Working in DMD, Other Neuromuscular Diseases

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Twenty-five research grants totaling more than $6.6 million have been awarded by the Muscular Dystrophy Association (MDA) to support studies into the mechanisms of neuromuscular diseases, including Duchenne and ... Read more

September 9, 2019September 9, 2019

BBP-418 in Development to Treat LGMD Type 2I, with Natural History Study to Start Soon

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BBP-418, an oral treatment candidate for patients with limb-girdle muscular dystrophy type 2I (LGMD2I), is being developed by ML Bio Solutions, a new subsidiary of BridgeBio Pharma, the company ... Read more

September 6, 2019September 6, 2019

September is MDA National Muscular Dystrophy Awareness Month

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As part of an effort to heighten awareness of its work in research, treatment and care — as well as to raise funds — the Muscular Dystrophy Association (MDA) ... Read more

September 6, 2019September 6, 2019

BioNews’ MD Forum Offers Support, Community – and a Place to Vent

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Online forums have markedly altered how patients, caregivers, and healthcare professionals communicate and connect. Even when miles apart, individuals can establish supportive relationships, share experiences and information, and sometimes ... Read more

September 4, 2019September 4, 2019

Vamorolone Improves Muscle Function in DMD Boys, Phase 2a Trial Data Show

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Vamorolone (VBP15), an experimental treatment for Duchenne muscular dystrophy (DMD) developed by ReveraGen Biopharma, improves muscle function of DMD patients in a dose-dependent manner, data from a Phase 2a extension ... Read more

August 30, 2019

‘Disorder’ Film Festival Offers Look at Siblings Touched by Rare Diseases

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Rare diseases deeply affect not only the children who experience them, but also their healthy brothers and sisters, as their parents can attest. Two entries in November’s “Disorder: ... Read more

August 30, 2019August 30, 2019

Jett Foundation’s Mission: ‘Making People Care About Duchenne’

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Many people are inspired by a son or brother with Duchenne muscular dystrophy (DMD). When it comes to Danielle Edwards, she’s literally made the disease her career. Edwards, 26, ... Read more

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