Muscular Dystrophy News

September 21, 2021September 21, 2021

Uplifting Athletes Tackles Funding for Rare Disease Research, Awareness

Football and science seem to be disparate fields of play at first glance, but the nonprofit Uplifting Athletes is finding common ground by leveraging the popularity of college gridiron games ... Read more

September 16, 2021September 16, 2021

BBP-418, Potential LGMD2i Therapy in Phase 2 Trial, on FDA Fast Track

Muscular Dystrophy News

BBP-418, an investigational disease-modifying medication that aims to improve muscle strength and function in people with limb-girdle muscular dystrophy type 2i (LGMD2i), has been given fast track designation by the U.S. ... Read more

September 15, 2021September 16, 2021

New Institute Aims to Leave No Rare Disease Patient Behind

Muscular Dystrophy News

A newly launched non-profit institute is seeking to advance research, and the development of new therapies, for people with rare diseases — a patient community with some of the largest ... Read more

September 14, 2021September 14, 2021

New Viral Vector Could Improve Gene Therapy for Muscle Diseases

Muscular Dystrophy News

A new viral vector that is better than conventional vectors — tools designed to deliver genetic material into cells — at targeting muscle cells may prove useful in developing a ... Read more

September 9, 2021September 9, 2021

Benitec Hoping to Start OPMD Gene Therapy Trial in US, EU Next Year

Muscular Dystrophy News

Benitec Biopharma is taking steps to get the needed regulatory clearances to start clinical testing in Europe and the U.S. of BB-301, its experimental gene therapy for oculopharyngeal muscular dystrophy ... Read more

September 8, 2021September 8, 2021

Group Focuses on Rare Disease Clinical Trial Participation

Muscular Dystrophy News

Participation in clinical trials exposes rare disease patients to financial, physical, and emotional pressures, according to the results of a patient focus group series. “Rare disease trial participants are running ... Read more

September 7, 2021September 7, 2021

$3M Grant to Help Build ‘Pediatric Cell Atlas of Skeletal Muscle’

Muscular Dystrophy News

A team of researchers received a $3-million grant from the Chan Zuckerberg Initiative to create a comprehensive blueprint of healthy skeletal muscle cells — whose dysfunction is associated with several ... Read more

September 2, 2021September 2, 2021

Score of MDA-led Activities Mark September, MD Awareness Month

Muscular Dystrophy News

A myriad of activities are afoot to mark National Muscular Dystrophy Awareness Month, observed each September. The events aim to raise funds for, and heighten awareness of, muscular dystrophy (MD) and ... Read more

September 1, 2021September 1, 2021

‘Running for Rare’: NORD a Charity Partner of 2021 NYC Marathon

Muscular Dystrophy News

The National Organization for Rare Disorders, known as NORD, was named an official charity partner of the 2021 TCS New York City Marathon, which will be held Nov. 7 both ... Read more

August 31, 2021August 31, 2021

AOC 1001 Earns Orphan Drug Status for DM1 in Europe

Muscular Dystrophy News

The European Commission (EC) has granted orphan designation to AOC 1001, Avidity Biosciences’ investigational therapy for myotonic dystrophy type 1 (DM1). This designation is given to investigational therapies that have ... Read more

August 26, 2021August 26, 2021

1st FDA-funded DMD Study Now Recruiting Patients to Test Ifetroban

Muscular Dystrophy News

Cumberland Pharmaceuticals is recruiting boys and men for a Phase 2 study evaluating the safety, efficacy, and duration of effect of its oral ifetroban therapy for cardiomyopathy — a disease ... Read more

August 25, 2021August 26, 2021

‘Not Alone in the Dark’: New Guide for, by Caregivers of Children With Rare Diseases

Muscular Dystrophy News

The National Alliance for Caregiving, in partnership with Global Genes, has issued a free guidebook, available online, that offers resources and support for caregivers of children with rare diseases. “The ... Read more

August 24, 2021August 25, 2021

Muscle MRI Scans Can Serve as Biomarker for DMD Progression

COVID-19 News, Muscular Dystrophy News

MRI scans of muscle can be used as a biomarker to assess disease progression in Duchenne muscular dystrophy (DMD) and to support the evaluation of different therapies in clinical trials, ... Read more

August 19, 2021August 19, 2021

DMD Adults Added to SSA Compassionate Allowances Program

Muscular Dystrophy News

The U.S. Social Security Administration (SSA) has added Duchenne muscular dystrophy (DMD) to its Compassionate Allowances (CAL) program list, accelerating the disabilities application process for adults with this genetic disorder. ... Read more

August 18, 2021August 18, 2021

Register Now for Global Genes’ RARE Patient Advocacy Summit

Muscular Dystrophy News

Registration is now open for Global Genes‘ 2021 RARE Patient Advocacy Summit. This year’s hybrid event will be livestreamed from California Sept. 27-29, and some seats also are available for attending ... Read more