MedPage Today on MSN

Antibody-Oligonucleotide Conjugate Shows Promise in Myotonic Dystrophy Type 1

Del-desiran reduced DMPK mRNA levels, but led to two serious adverse events ...
kristv

Local mother advocates for muscular dystrophy disease with 'Fill the Boot' Campaign

is your neighborhood reporter for North Beach, Downtown Corpus Christi and the Bay Area. CORPUS CHRISTI, Texas — A local mother is raising awareness for Duchenne muscular dystrophy (DMD) as her son ...
NJ.com

What is muscular dystrophy, the rare genetic disease Gilbert Gottfried had?

Tributes flooded the internet for Gilbert Gottfried after his family shared on Tuesday via Twitter that he died following “a long illness.” Since then, there has been more awareness around muscular ...
Science Daily

Mouse model reveals liver involvement in muscular dystrophy

A new mouse model mimicking the liver symptoms of myotonic dystrophy type 1 -- the most prevalent form of adult-onset muscular dystrophy -- provides insight into why patients develop fatty liver ...

Muscular Dystrophy Association Announces Łukasz Sznajder, PhD, MSc, to Receive Inaugural MDA Research Momentum Award

This award honors an emerging leader shaping the future of neuromuscular research will be presented at the 2026 MDA Clinical ...

Nationwide MDA Shamrocks Campaign Advances Independence for People Living with Rare Neuromuscular Conditions

The Muscular Dystrophy Association (MDA) announced its nationwide MDA Shamrocks campaign, uniting with retail partners across the country to make a meaningful impact for families living with rare ...
CNN

FDA advisers narrowly vote in favor of experimental gene therapy for rare muscle disease

Most parents wouldn’t be thrilled with the idea of their kids getting hooked up to an IV bag filled with trillions of viruses. But for Melanie Hennick, whose son, Connor, has Duchenne muscular ...
Medical Xpress

Disease or Syndrome: Muscular Dystrophy, Duchenne

If cancer is a disease of overabundance, where cells divide without restraint and tumors grow despite the body's best interests, then degenerative diseases are disorders of deprivation. When ...
The American Journal of Managed Care

Duchenne Muscular Dystrophy

Delandistrogene moxeparvovec, a gene therapy for Duchenne muscular dystrophy (DMD), showed high dystrophin expression and a favorable safety profile in young patients. Delandistrogene moxeparvovec ...
The American Journal of Managed Care

Regenerating Neurons, Muscle, and Hope in the Field of Muscular Dystrophy

The MDA's 75th anniversary conference highlights advancements in gene therapy, precision medicine, and patient-centered care for muscular dystrophy. Discussions focus on equitable access to ...
Business Wire

Promising disease modifying approach to Duchenne muscular dystrophy with Neu-REFIX® Beta 1,3-1,6 glucan* from Japan; the first such clinical report.

TOKYO--(BUSINESS WIRE)--Duchenne Muscular Dystrophy (DMD) patients showed signs of disease progress slowing down, after oral consumption of Neu REFIX ß-glucan for 45 days along with routine ...

Bullish analyst opinion on Dyne (DYNE) follows rare disease drug progress in Japan

Dyne Therapeutics Inc. (NASDAQ:DYN) is one of the 13 hot stocks to buy with the highest upside potential. As of February 13, ...