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).

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

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

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

Novel drug holds promise for treating Duchenne muscular dystrophy

A novel drug holds promise for treating Duchenne muscular dystrophy (DMD), a rare genetic disorder that causes severe muscle degeneration.

16 Dec 2024

Harnessing exosomes to deliver therapeutic proteins

Researchers at The Ottawa Hospital and the University of Ottawa have discovered an 18-digit code that allows proteins to attach themselves to exosomes - tiny pinched-off pieces of cells that travel around the body and deliver biochemical signals.

11 Dec 2024

SRF awards $230,000 grant to advance research on SYNGAP1-Related Disorders

The SynGAP Research Fund (SRF) has awarded a $230,000 grant to The Jackson Laboratory Rare Disease Translational Center to characterize a mouse model for SYNGAP1-Related Disorders, (SRD), a severe neurodevelopmental disorder marked by seizures, intellectual disabilities, and motor and behavioral challenges.

4 Dec 2024

StitchR technology delivers large genes for muscular dystrophy treatment

Gene therapy can effectively treat various diseases, but for some debilitating conditions like muscular dystrophies there is a big problem: size.

14 Nov 2024

CRISPR-Cas13 emerges as a game changer in RNA-targeted therapies

In recent years, the scientific community has made significant strides in the field of gene editing, particularly through the development of the CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) systems.

25 Sep 2024

New tool shows promise for assessing neurobehavioral difficulties in children with Duchenne muscular dystrophy

A pilot study using a new screening tool shows promise for assessing neurobehavioral difficulties in children with Duchenne muscular dystrophy (DMD) and improving quality of life for patients and their families.

14 Aug 2024

Breakthrough gene therapy restores dystrophin protein in Duchenne muscular dystrophy

Indiana University School of Medicine researchers have made a significant breakthrough in developing a new gene therapy approach that restores full-length dystrophin protein, which could lead to new treatments for people with Duchenne muscular dystrophy (DMD).

24 Jul 2024

UH research identifies key mechanisms of skeletal muscle regeneration

Newly published research from the University of Houston College of Pharmacy identifies key mechanisms of skeletal muscle regeneration and growth of muscles following resistance exercise.

22 Jul 2024

Breakthrough gene therapy offers hope for Duchenne muscular dystrophy

A new gene therapy treatment for Duchenne muscular dystrophy shows promise of not only arresting the decline of the muscles of those affected by this inherited genetic disease, but perhaps, in the future, repairing those muscles.

18 Jul 2024

Enzymatic RNA synthesis paves the way for sustainable therapeutics

While the COVID-19 vaccines introduced many people to RNA-based medicines, RNA oligonucleotides have already been on the market for years to treat diseases like Duchenne Muscular Dystrophy and amyloidosis.

12 Jul 2024

New therapeutic approach for Duchenne muscular dystrophy combines LED therapy with idebenone

In an animal model of Duchenne muscular dystrophy, Brazilian researchers tested a therapy that combines photobiomodulation using laser light or light-emitting diodes (LEDs) with idebenone, an antioxidant compound investigated for application in neurodegenerative diseases.

14 Jun 2024

Harnessing immune enhancement to combat drug-resistant tuberculosis

Experts are working on novel immune-enhancing therapies called host-directed therapies to use the body's own immune system to target tuberculosis, with hopes that they could tackle even the drug-resistant forms of the disease.

26 Apr 2024

Adeno-associated virus: The gene therapy revolution faces manufacturing and safety hurdles

Recombinant adeno-associated virus (rAAV)-based genetic applications to treat human diseases.

5 Apr 2024

Lurie Children's Hospital administers first gene therapy for Duchenne muscular dystrophy in Illinois

On March 27, 2024, Ann & Robert H. Lurie Children's Hospital of Chicago treated its first patient with ELEVIDYS (delandistrogene moxeparvovec-rokl), the first gene therapy for Duchenne muscular dystrophy – a rare, genetic disease characterized by progressive muscle damage and weakness.

3 Apr 2024

Breakthrough in CRISPR delivery promises safer gene editing

Recent research in PNAS highlights advancements in non-viral, cell-specific delivery methods for CRISPR-Cas systems, emphasizing their potential to enhance research and gene therapy applications by minimizing risks associated with off-target effects and improving targeted delivery.

7 Mar 2024