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.

Gene therapy helps build muscle mass and reduces severity of osteoarthritis in mice

Exercise and physical therapy often are recommended to help people who have arthritis. Both can strengthen muscle -; a benefit that also can reduce joint pain.

8 May 2020

Scripps professor receives Research Program Award for advancing RNA-targeting medicines

In recognition of his high-impact work advancing the field of RNA-targeting medicines, the National Institute of Neurological Disorders and Stroke (NINDS), part of the National Institutes of Health, has awarded Scripps Research Chemistry Professor Matthew Disney, PhD, a prestigious Research Program Award, to aid Disney's development of treatments for incurable diseases such as Alzheimer's, Parkinson's, ALS and frontotemporal dementia.

4 May 2020

New study indicates novel therapies for Duchenne muscular dystrophy

When an actor is unable to perform in the theatre, an understudy--ideally one with some practice in the role--can take her place on stage.

25 Apr 2020

Temple researchers reverse muscle fibrosis associated with overuse injury in animals

Overuse injuries - think muscle strains, tennis elbow, and rotator cuff tears - are a considerable problem in the United States, especially among young athletes.

30 Mar 2020

Model that sheds light on severe Duchenne muscular dystrophy could pave the way for new therapies

Researchers have developed an experimental model of severe Duchenne muscular dystrophy that offers insight into the disease.

26 Mar 2020

Finding new treatments for muscular dystrophy with CRISPR-Cas9

A new study out of Boston's Children's Hospital has used the gene-editing tool CRISPR-Cas9 to explore the fatal genetic condition called facioscapulohumeral dystrophy (FSHD, one of the family of muscular dystrophies), and to test out the potential utility of various genes involved in this disorder. The research is published in the journal Science Translational Medicine.

25 Mar 2020

New form of gene editing slows ALS progression in mice

With a new CRISPR gene-editing methodology, scientists from the University of Illinois at Urbana-Champaign inactivated one of the genes responsible for an inherited form of amyotrophic lateral sclerosis - a debilitating and fatal neurological disease for which there is no cure.

26 Feb 2020

A promising new oral therapy to speed up healing of broken bones

People with diabetes are at a higher risk of fracturing a bone than the general population. And if they do break one it also takes longer than normal to heal.

24 Feb 2020

Small molecules may open the door to new therapies for Duchenne muscular dystrophy

Researchers identified a group of small molecules that may open the door to developing new therapies for Duchenne muscular dystrophy, an as-yet-uncured disease that results in devastating muscle weakening and loss.

24 Feb 2020

Study reveals molecular pathogenesis of muscular dystrophy-associated cardiomyopathy

In a study recently published in Nature Communications scientists at Okayama University describe the detailed molecular pathogenesis of muscular dystrophy-associated cardiomyopathy in mice lacking the fukutin gene (Fktn), the causative gene for Fukuyama muscular dystrophy.

13 Feb 2020

Simple accelerometers improve diagnosis of Duchenne muscular dystrophy

Duchenne muscular dystrophy is the most common type of muscular dystrophy, affecting more than 10,000 males at birth per year in the United States with severe physical disability, chronic wasting and muscle deterioration.

11 Feb 2020

Study uncovers molecular mechanism that triggers a rare type of muscular dystrophy

A study led by the IBB-UAB has identified the molecular mechanism through which a protein, when carrying genetic mutations associated with a rare disease known as limb-girdle muscular dystrophy, type 1G, accelerates its tendency to form amyloid fibrils and finally triggers the appearance of the disease.

29 Jan 2020

New gene correction therapy for hereditary muscular disease among children

Duchenne type muscular dystrophy is the most common hereditary muscular disease among children, leaving them wheelchair-bound before the age of twelve and reducing life expectancy.

27 Jan 2020

Asian parents with terminally ill children may not share psychological pain with their spouses

A study of families in Singapore with terminally ill children found that parents tend to defer discussing their psychological pain with their spouses to protect them from emotional distress.

16 Jan 2020

PerkinElmer provides newborn screening assay for PPMD’s New York State pilot program

PerkinElmer, Inc., a global leader committed to innovating for a healthier world, today announced that it is providing the newborn screening assay for Parent Project Muscular Dystrophy’s Newborn Screening Pilot for Duchenne Muscular Dystrophy (Duchenne).

From Revvity 14 Jan 2020

Horizon Discovery to offer novel base editing technology for clinical research

Horizon Discovery today announced that it will provide access to a novel base editing technology licensed from Rutgers for exclusive use in therapeutic, diagnostic and services applications.

From Revvity 14 Jan 2020

Researchers find key to preventing muscular dystrophy-related heart disease

A Rutgers-led team may have found the key to preventing Duchenne muscular dystrophy (DMD)-related heart disease, the leading cause of death in patients living with the disease.

14 Jan 2020

First in vitro system can help study early spine development in humans

More than 20 years ago, the lab of developmental biologist Olivier Pourquié discovered a sort of cellular clock in chicken embryos where each "tick" stimulates the formation of a structure called a somite that ultimately becomes a vertebra.

9 Jan 2020

Single-particle method helps determine isoelectric point of viruses

Viruses are not easy to characterize. But we need to because being able to quickly predict the surface charge of viruses opens up new possibilities for vaccine purification and making gene therapy treatments for eye diseases and muscular dystrophy.

8 Jan 2020

MDI Biological Laboratory announces expansion of innovative data literacy program

The MDI Biological Laboratory has announced that it is expanding its innovative SEPA (Science Education Partnership Award) program, which aims to improve secondary school education in data literacy while addressing the public health threat posed by arsenic in well water.

3 Jan 2020