Muscular Dystrophy News

June 21, 2022June 21, 2022

EDG-5506 Safely Lowering Muscle Damage Markers in Becker MD Trial

The experimental oral therapy EDG-5506 has been generally well-tolerated and lowered markers of muscle damage among men with Becker muscular dystrophy (BMD) in the open-label ARCH clinical trial, early data show. ... Read more

June 16, 2022June 17, 2022

Defeat Duchenne Canada Grants $1M to DMD Treatment Research

Muscular Dystrophy News

Defeat Duchenne Canada is investing $1.14 million in three research projects aimed at providing better treatments for Duchenne muscular dystrophy, the most common type of muscular dystrophy and one that ... Read more

June 14, 2022June 15, 2022

Fundraiser Games Event for MDA Goes Live on Twitch This Saturday

Muscular Dystrophy News

The Muscular Dystrophy Association (MDA) is hosting a one-day games streaming fundraiser, dubbed “MDA Rivals,” this Saturday, June 18. The event, streaming on MDA’s Twitch channel, will feature stars from ... Read more

June 9, 2022June 10, 2022

FibroGen Completes Enrollment for Second Phase 3 Trial of Pamrevlumab

Muscular Dystrophy News

FibroGen has completed patient enrollment for its Phase 3 trial evaluating pamrevlumab in combination with corticosteroids as a potential treatment for children with Duchenne muscular dystrophy (DMD). The LELANTOS-2 study (NCT04632940) ... Read more

June 7, 2022June 7, 2022

Diuretics, BNP Levels Predict Survival in Late-stage DMD: Study

Muscular Dystrophy News

Reduced heart function is associated with a higher rate of mortality among people with Duchenne muscular dystrophy (DMD), a new study reports. Results, however, did not suggest that reduced heart ... Read more

June 2, 2022June 2, 2022

Review Identifies 23 Factors Affecting Duchenne MD Prognosis

Muscular Dystrophy News

Treatment with glucocorticoids and heart medicines are key factors in determining the prognosis of Duchenne muscular dystrophy (DMD), according to a study. Results also highlighted genetics as an important factor ... Read more

May 31, 2022May 31, 2022

DMD Diagnosis Takes as Long Today as Decades Ago, Study Finds

Muscular Dystrophy News

Despite broad initiatives seeking improvements, the time it takes an individual with Duchenne muscular dystrophy (DMD) to get a correct diagnosis has remained largely unchanged over the past three decades, ... Read more

May 26, 2022May 26, 2022

Tamoxifen Increases Survival of DMD Heart Muscle in Cell Study

Muscular Dystrophy News

Treatment with the breast cancer medicine tamoxifen improved the functionality and survival of heart muscle cells in a cellular model of Duchenne muscular dystrophy (DMD), a study reports. The study, “Tamoxifen ... Read more

May 24, 2022May 25, 2022

FSHD Society, C-Path Gathering Trial Data on Placebo Group Patients

Muscular Dystrophy News

The FSHD Society has joined forces with the Critical Path Institute (C-Path) to facilitate the collection of data from people with facioscapulohumeral muscular dystrophy (FSHD) who were given a placebo ... Read more

May 19, 2022May 20, 2022

More Data Support Gene Therapy for DMD Exon Duplications

Muscular Dystrophy News

New data from a Phase 1/2 clinical trial of an exon-skipping gene therapy suggest it may help preserve muscle function if delivered early on to Duchenne muscular dystrophy (DMD) patients ... Read more

May 17, 2022May 17, 2022

Elamipretide Wins FDA Orphan Drug Designation to Treat DMD

Muscular Dystrophy News

The U.S. Food and Drug Administration (FDA) has granted orphan drug status to elamipretide for treating Duchenne muscular dystrophy (DMD). According to Stealth BioTherapeutics, the therapy’s developer, elamipretide has been found to ... Read more

May 12, 2022May 12, 2022

Center Is First in State to Win PPMD Certification for Duchenne Care

Muscular Dystrophy News

The Spectrum Health Helen DeVos Children’s Hospital (HDVCH) in Michigan has been recognized as providing the highest quality of care for patients with Duchenne muscular dystrophy. Located in Grand Rapids, ... Read more

May 10, 2022May 10, 2022

Novel Exon-skipping Therapy Mix May Be Able to Treat Over 40% of DMD

Muscular Dystrophy News

An international team of scientists has developed a novel exon-skipping therapy mixture that has the potential to treat more than 40% of people with Duchenne muscular dystrophy (DMD). The therapy ... Read more

May 5, 2022May 6, 2022

MDA Campaigns Support Summer Camp, NMD Community

COVID-19 News, Muscular Dystrophy News

To help support its popular summer camp and the neuromuscular disease community, the Muscular Dystrophy Association (MDA) has launched campaigns through October that involve more than 1,000 retailers nationwide. Proceeds ... Read more

May 3, 2022May 3, 2022

Phase 3 Trial of Pfizer’s Gene Therapy Expected to Open in US in June

Muscular Dystrophy News

The U.S. Food and Drug Administration (FDA) has given Pfizer the go-ahead to open U.S. sites for the Phase 3 CIFFREO clinical trial, testing the experimental gene therapy PF-06939926 in boys with Duchenne muscular ... Read more