Duchenne Muscular Dystrophy News and Research

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Duchenne muscular dystrophy (DMD) is a progressive muscle disorder that causes the loss of both muscle function and independence. DMD is perhaps the most prevalent of the muscular dystrophies and is the most common lethal genetic disorder diagnosed during childhood today. Each year, approximately 20,000 children worldwide are born with DMD (one of every 3,500 male children).

Prednisone may impair bone health in children with Duchenne muscular dystrophy

In a new study published in Scientific Reports, an international and multidisciplinary research team including Binghamton University researchers identified a set of blood proteins that may explain why the corticosteroid prednisone, although beneficial in treating Duchenne muscular dystrophy (DMD), can interfere with childhood growth and bone health.

4 Sep 2026

Generative AI helps scientists design entirely new RNA transporters

Researchers at Helmholtz Munich and TUM have developed Synthetic Transfer Vehicles (STVs), an entirely new class of RNA transporters.

2 Sep 2026

Advances in X chromosome inactivation open therapeutic opportunities for X-linked genetic disorders

A new review explores how advances in understanding X chromosome inactivation (XCI) are creating potential therapeutic opportunities for a range of X-linked genetic disorders, including Rett syndrome, Fabry disease, Duchenne muscular dystrophy, hemophilia, and others.

3 Aug 2026

TRF2 protein preserves muscle stem cell identity and regeneration

A protein best known for protecting the ends of chromosomes also helps muscle stem cells preserve their identity and repair damaged tissue, according to a new study from researchers at the Perelman School of Medicine at the University of Pennsylvania.

31 Jul 2026

Deramiocel cell therapy slows muscle and heart damage in advanced DMD

A cell therapy called deramiocel could slow muscle weakening in boys and young men with advanced Duchenne muscular dystrophy (DMD), and may also slow heart damage in those who already have heart muscle disease, a phase 3 clinical trial published in The Lancet has found.

29 Jul 2026

NOX4 inhibition may protect hearts affected by Duchenne muscular dystrophy

Researchers at the University of South Florida USF Health Morsani College of Medicine have identified a potential pathway that could protect cardiac function in people with Duchenne muscular dystrophy, a progressive and fatal genetic disease that weakens the body's muscles.

17 Jul 2026

New treatment platform delivers full-length mRNA for Duchenne muscular dystrophy

A new treatment platform developed by researchers at The University of Texas MD Anderson Cancer Center was able to deliver messenger RNA (mRNA) of the full-length DMD gene into preclinical models of Duchenne muscular dystrophy, successfully restoring the production of an important muscle protein, dystrophin, and dramatically improving muscle strength, endurance and function in vivo.

11 Jun 2026

New ‘heart-on-a-chip’ to halt cardiac damage caused by Duchenne

Duchenne Parent Project Spain (DPPE) has launched the BEAT Project, a research initiative focused on one of the key challenges associated with Duchenne and Becker muscular dystrophies: progressive heart damage. Cardiomyopathy is the leading cause of death in patients with DMD.

7 May 2026

New collaboration targets muscle loss treatments for space missions

The Center for Myokine Convergence Research at Korea University College of Medicine (Director: Professor Hyeon Soo Kim, Department of Anatomy, Korea University College of Medicine) has signed a memorandum of understanding (MOU) with MFC to jointly develop therapeutics for muscle loss in astronauts.

10 Apr 2026

Oz escalates Medicaid fraud claims against states after focus on Minnesota

The Trump administration has signaled a willingness to halt billions of dollars in federal health payments to multiple states, mirroring moves they made against Minnesota.

20 Mar 2026

New BIND screener identifies brain-related comorbidities risk in Duchenne muscular dystrophy

In research published in Developmental Medicine & Child Neurology, investigators developed a brief, reliable, and valid screening tool to help identify individuals with Duchenne muscular dystrophy (a neuromuscular disorder) who are at increased risk of brain-related comorbidities, such as language disorders, attention-deficit/hyperactivity disorder, and anxiety.

14 Jan 2026

New method yields up to twice as many therapeutic myogenic cells as previous protocols

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.

30 Oct 2025

Under Trump, FDA seeks to abandon expert reviews of new drugs

FDA leaders under President Donald Trump are moving to abandon a decades-old policy of asking outside experts to review drug applications, a move critics say would shield the agency's decisions from public scrutiny.

12 Sep 2025

Improving gene therapy safety with human kidney organoids

Ryuji Morizane, MD, PhD, of the Department of Medicine at Massachusetts General Hospital, is the senior/corresponding author of a new paper published in Signal Transduction and Targeted Therapy, "AAV for gene therapy drives a nephrotoxic response via NFκB in kidney organoids."

19 Aug 2025

Researchers solve longstanding mystery of protein release in cells

It's known as biology's central dogma: All living organisms' genetic information is stored in DNA, which is transcribed into RNA, which is translated into proteins that perform nearly all essential tasks in a cell.

5 Aug 2025

Marbling of fat inside muscles found to block healing and weaken strength

Consider the delicate web of fat in a Wagyu steak. The "marbling" that makes carnivore connoisseurs swoon is a visual heuristic for quality flavor.

28 Jul 2025

Targeting GLUD1 shows promise in restoring muscle function in Duchenne muscular dystrophy

New research has identified the enzyme glutamate dehydrogenase 1 (GLUD1) as a new therapeutic target for Duchenne muscular dystrophy (DMD).

17 Jul 2025

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

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

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