Muscular Dystrophy News and Research

RSS

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.

Advances in chemistry and delivery are taking oligonucleotide medicines beyond rare diseases

A review in Cell Reports Medicine examines how advances in oligonucleotide chemistry and delivery have established ASOs and RNA interference therapies as a class of genetic medicines, with 21 oligonucleotides approved at the time of writing. It also identifies extrahepatic delivery, safety, target validation, individualized treatment, and data quality as priorities as the field expands into common diseases and explores AI-assisted development.

24 Sep 2026

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

Stanford scientists develop an ultracompact gene activation tool

The ability to precisely adjust the expression of genes inside the human body - ramping up protective ones and tamping down harmful ones - holds enormous potential for treating and preventing disease.

28 Aug 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

New gene editing tool reduces Huntington's symptoms in mice

A gene editing tool designed to precisely rewrite the gene that causes Huntington's disease reduced toxic protein fragments and symptoms associated with the disease in mice, researchers at the University of Illinois Urbana-Champaign report.

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

Myotonia may play a much larger role in driving muscle damage than previously recognized

For decades, researchers studying myotonic dystrophy type 1 (DM1) have focused on the disease's underlying genetic cause: a mutation that produces a toxic form of RNA, disrupting the normal processing of thousands of genetic messages inside cells.

13 Jul 2026

Open source tool automates data reanalysis to detect rare diseases

A newly developed open-source tool designed for rigorous reanalysis of genomic data is highly effective at detecting new rare disease diagnoses.

24 Jun 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

Study reveals protein driving fast-twitch muscle formation

University of Maine researchers have published new findings about how muscles form, why certain muscle diseases develop and why symptoms may not appear until years after muscle degeneration begins.

8 Jun 2026

Hidden junk DNA could play decisive role in cancer

Many repetitive regions of the genome have been considered "junk DNA" because the technologies available did not allow them to be studied at sufficient resolution.

5 Jun 2026

Advancing Human Muscle Models for Drug Development and Preclinical Testing

Transforming drug development, Myomaker Bio's lab-grown muscle platforms offer human-relevant models for effective preclinical testing and risk reduction.

From Myomaker Bio 2 Jun 2026

New ultrasound pacemaker design promises minimal recovery time for cardiac patients

MIT engineers have developed a noninvasive pacemaker that stimulates the heart using ultrasound. The design could one day provide a surgery-free alternative to traditional cardiac implants.

2 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

Yale researchers use zebrafish to find precision treatments for autism

In recent decades, the zebrafish has become one of the most valuable model organisms in scientific research.

8 Apr 2026

Toxic RNA drives progressive heart damage in myotonic dystrophy

Myotonic dystrophy type 1 (DM1) is the most common cause of adult-onset muscular dystrophy, a genetic disorder that leads to muscle weakness and wasting, but also affects the brain, the gastrointestinal tract and the heart.

3 Apr 2026