Muscular Dystrophy News and Research

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The muscular dystrophies (MD) are a group of more than 30 genetic diseases characterized by progressive weakness and degeneration of the skeletal muscles that control movement. Some forms of MD are seen in infancy or childhood, while others may not appear until middle age or later. The disorders differ in terms of the distribution and extent of muscle weakness (some forms of MD also affect cardiac muscle), age of onset, rate of progression, and pattern of inheritance.

New cellular entry pathway improves gene therapy outcomes

Scientists from the Centenary Institute and the University of Sydney have made a landmark discovery that could lead to safer and more effective gene therapies for a range of serious genetic disorders including Duchenne muscular dystrophy, Pompe disease and hemophilia.

15 Jul 2025

Uncovering the hidden architecture of the genome in three dimensions

In biology textbooks and beyond, the human genome and DNA therein typically are taught in only one dimension. While it can be helpful for learners to begin with the linear presentation of how stretches of DNA form genes, this oversimplification undersells the significance of the genome's 3D structure.

12 Jul 2025

Improving RNA drug delivery through intracellular traffic control

A recent study involving researchers from the University of Basel reveals that slowing down the intracellular transport of RNA-based drugs can significantly enhance their effectiveness.

1 Jul 2025

Researchers reverse muscle stem cell aging with Prostaglandin E2

The population across developed countries is getting older and the associated frailty and debilitation are becoming major health problems.

13 Jun 2025

Experimental ALS drug shows unprecedented recovery in some patients

When Columbia neurologist and scientist Neil Shneider speaks to his ALS patients who volunteer for experimental therapies, he's unwaveringly honest.

23 May 2025

AAN releases Evidence in Focus Article on new gene therapy for Duchenne muscular dystrophy

To help neurologists, clinicians and families understand the current evidence for a new gene therapy for Duchenne muscular dystrophy called delandistrogene moxeparvovec, the American Academy of Neurology (AAN) has issued an Evidence in Focus article, published May 14, 2025, online in Neurology.

14 May 2025

Estrogen-related receptors could be a key to repairing energy metabolism and muscle fatigue

A new Salk Institute study suggests estrogen-related receptors could be a key to repairing energy metabolism and muscle fatigue.

12 May 2025

Existing therapies may help control chronic muscle inflammation in Duchenne muscular dystrophy

A new study led by researchers at Hospital for Special Surgery (HSS) suggests that currently available therapies may help control chronic muscle inflammation in Duchenne muscular dystrophy (DMD), a severe condition that leads to muscle weakness and premature death.

30 Apr 2025

Antimalarial drug enhances effect of aminoglycosides in genetic disorders

The antimalarial drug mefloquine could help treat genetic diseases such as cystic fibrosis, Duchenne muscular dystrophy, as well as some cancers.

23 Apr 2025

Researchers uncover genetic link between autism spectrum disorder and myotonic dystrophy type 1

Scientists from The Hospital for Sick Children (SickKids) and University of Las Vegas Nevada (UNLV) have uncovered a genetic link between autism spectrum disorder (ASD) and a rare genetic condition called myotonic dystrophy type 1 (DM1).

21 Apr 2025

Trump’s DEI undoing undermines hard-won accommodations for disabled people

For years, White House press conferences included sign language interpreters for the deaf.

3 Apr 2025

New protein target could aid in muscle regeneration for degenerative conditions

For millions of people, losing muscle isn't just about weakness; it's about losing independence. Whether caused by Duchenne muscular dystrophy, aging or other degenerative conditions, muscle loss can make everyday activities – like walking, climbing stairs or even standing up – a daily struggle

26 Mar 2025

Many people with disabilities risk losing their Medicaid if they work too much

Zach Mecham has heard politicians demand that Medicaid recipients work or lose their benefits. He also has run into a jumble of Medicaid rules that effectively prevent many people with disabilities from holding full-time jobs.

25 Mar 2025

Testing a novel approach to restore normal heart function in Myotonic Dystrophy Type 1

Myotonic Dystrophy Type 1 (DM1) is the most common adult-onset form of muscular dystrophy and a condition that severely affects multiple organs including skeletal muscle, heart, brain and the gastrointestinal tract.

18 Mar 2025

Research links kidney dysfunction to muscle weakness in myotonic dystrophy type 1

Thurman M. Wheeler, MD, of the Department of Neurology at Massachusetts General Hospital, is the senior author of a paper published in Nature Communications, "Analysis of human urinary extracellular vesicles reveals disordered renal metabolism in myotonic dystrophy type 1."

8 Mar 2025

Protein cluster discovery could lead to muscular dystrophy treatments

A new discovery about how tiny protein clusters form in cells could pave the way for treatments for Emery-Dreifuss muscular dystrophy (EDMD), a rare genetic disorder that causes muscle weakness and heart problems.

6 Mar 2025

Study sheds light on the role of fibro-adipogenic progenitors in muscle regeneration and degeneration

A recent study has shed new light on the role of fibro-adipogenic progenitors (FAPs) in muscle regeneration, fibrosis, and degeneration.

5 Mar 2025

New mRNA technique targets disease cells while sparing healthy ones

Imagine a breakthrough in cancer treatment where only malignant cells are targeted, sparing healthy host cells; or patients with abnormal protein synthesis are treated to produce a healthy protein.

19 Feb 2025

Communication between body muscle and pituitary gland may play unexpected role in female fertility

A newly discovered line of communication between body muscle and the pituitary gland may play an unexpected role in female fertility, according to a new study.

6 Feb 2025

SPLICER shows promise in Alzheimer's and other diseases

A new gene editing tool that helps cellular machinery skip parts of genes responsible for diseases has been applied to reduce the formation of amyloid-beta plaque precursors in a mouse model of Alzheimer's disease, researchers at the University of Illinois Urbana-Champaign report.

23 Dec 2024