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.

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

SpaceX CRS-31 supports heart health, neurodegeneration, and student science

More than 25 payloads sponsored by the International Space Station (ISSInternational Space Station) National Laboratory, including technology demonstrations, in-space manufacturing, student experiments, and multiple projects funded by the U.S. National Science Foundation (NSF), are bound for the orbiting outpost.

4 Nov 2024

New insights into cardiac defects in Myotonic Dystrophy Type 1

People with Myotonic Dystrophy Type 1 (DM1), the most common adult-onset form of muscular dystrophy, progressively lose muscle mass and strength in their lower legs, hands, neck and face.

29 Oct 2024

More restrooms have adult-size changing tables to help people with disabilities

The blue-and-white highway sign for the eastbound rest stop near here displays more than the standard icon of a person in a wheelchair, indicating facilities are accessible to people who can't walk.

3 Oct 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

Understanding Global Harmonization of Regulatory CMC within Veterinary Medicine with Tammy Newbold

In this interview, News Med talks to Tammy Newbold about Global Harmonization of Regulatory CMC within Veterinary Medicine.

From DSI, a PLG Company 26 Jul 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

Study sheds light on the disease development of Facioscapulohumeral Muscular Dystrophy

A recent study has found that the SMCHD1 protein plays a key role in controlling how genes are processed, which affects the progression of Facioscapulohumeral Muscular Dystrophy (FSHD).

4 Jun 2024

Unraveling the mechanisms of PMP22 duplication in Charcot-Marie-Tooth disease

Charcot-Marie-Tooth disease (CMT), a group of heritable disorders that affect the peripheral nerves, is marked by specific genetic changes.

4 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