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.

Mutations responsible for common inherited heart condition corrected using CRISPR-Cas9 therapy

Using the CRISPR-Cas9 gene editing system, UT Southwestern researchers corrected mutations responsible for a common inherited heart condition called dilated cardiomyopathy (DCM) in human cells and a mouse model of the disease.

21 Dec 2022

Research estimates the evolutionary origins of functional human microgenes

Modern humans evolutionarily split from our chimpanzee ancestors nearly 7 million years ago, yet we are continuing to evolve.

20 Dec 2022

Tiny messenger can be promising candidate for treatment of diseases associated with muscular atrophy

WNT messenger proteins are crucial for a variety of cellular functions. A specific WNT messenger can increase muscle mass and improve regeneration in skeletal muscle.

14 Dec 2022

Scientists develop RNA-targeting strategy to repair genetic cause of ALS and dementia

Scientists at UF Scripps Biomedical Research have developed a potential medicine for a leading cause of ALS and dementia that works by eliminating disease-causing segments of RNA. The

22 Nov 2022

Researchers receive $9 million grant to study how ‘jumping genes’ influence Alzheimer's disease

Scientists have identified a handful of gene mutations that cause or contribute to the onset of Alzheimer's disease.

11 Oct 2022

New discovery changes the understanding of Duchenne muscular dystrophy

Duchenne muscular dystrophy (DMD) is caused by a genetic mutation and affects one in every 5,000 boys born. Because the affected gene is on the X chromosome, girls are carriers of the mutant gene but develop the disease only very rarely (one in about 50 million).

26 Sep 2022

Experimental drug prolongs survival, improves muscle function in mice severe Duchenne muscular dystrophy

Researchers at Johns Hopkins Medicine report that an experimental drug first developed to treat kidney disease prolongs survival and improves muscle function in mice genetically engineered to develop a severe form of Duchenne muscular dystrophy (DMD).

13 Sep 2022

Researchers receive $1.2 million Keck Foundation grant to find molecules for targeting the 'undruggable proteome'

The W.M. Keck Foundation has awarded a $1.2 million grant to medicinal chemistry and computational biology researchers at the University of Kansas and the University of Chicago focused on addressing a long-unresolved problem in biomedical research -; finding molecules able to target the "undruggable proteome."

29 Aug 2022

Cancer drug helped delay progression of Duchenne muscular dystrophy in mice

Researchers at UBC’s School of Biomedical Engineering have discovered that an existing cancer drug could have potential as a treatment for muscular dystrophy.

1 Jul 2022

Existing cancer drug shows promise for treating patients with muscular dystrophy

Researchers at the University of British Columbia's School of Biomedical Engineering have discovered that an existing cancer drug could have potential as a treatment for muscular dystrophy.

29 Jun 2022

NUS researchers discover a new way to interpret unsolved Mendelian diseases

The development of an embryo is a well-orchestrated string of processes, ensuring correct formation and positioning of vital organs of the growing organism.

29 Jun 2022

U-M researchers reveal how a mutated protein acts as risk factor in Parkinson’s disease

In about a fifth of the cases of Parkinson’s disease, look to a small, malfunctioning protein in the lysosome as a risk factor, say University of Michigan researchers.

23 Jun 2022

The promise of RNA medicine is targeted and personalized

The development of messenger RNA (mRNA) vaccines against the severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) has only increased the impetus in this field.

11 May 2022

Study supports daily steroid doses for children with Duchenne muscular dystrophy

New research published in JAMA recommends daily steroid doses for children with Duchenne muscular dystrophy (DMD), marking a significant change in how the disease is treated.

4 May 2022

DGP student investigates sexual dysmorphism in mice

Isabella Salamone is an eighth-year student in the Driskill Graduate Program in Life Sciences (DGP) who is studying sexual dysmorphism in mice in the laboratory of Elizabeth McNally, MD, PhD, the Elizabeth J. Ward Professor of Genetic Medicine and director of the Center for Genetic Medicine.

28 Apr 2022

Researchers develop new platform capable of mitochondrial DNA editing

Researchers from the Center for Genome Engineering within the Institute for Basic Science developed a new gene-editing platform called transcription activator-like effector-linked deaminases, or TALED.

25 Apr 2022

Lab grown, self-renewing muscle stem cells can repair muscle tissue damage in mice

In proof-of-concept experiments, Johns Hopkins Medicine scientists say they have successfully cultivated human muscle stem cells capable of renewing themselves and repairing muscle tissue damage in mice, potentially advancing efforts to treat muscle injuries and muscle-wasting disorders in people.

20 Apr 2022

Regular cycling can improve mobility in patients with myotonic dystrophy

A study led by McMaster University researchers has found that regular cycling can greatly improve mobility in patients with myotonic dystrophy (MD), a genetic disease that causes muscle degeneration.

11 Apr 2022

Study shows the benefits of once-weekly prednisone in obese mice

Obese mice that were fed a high-fat diet and that received prednisone one time per week had improved exercise endurance, got stronger, increased their lean body mass and lost weight, reports a new Northwestern Medicine study published in The Journal of Experimental Medicine.

1 Apr 2022

Using CRISPR/Cas to develop biosafety materials

Researchers discuss the use of the highly efficient CRISPR/Cas technologies to identify viral genome sequences for biosafety purposes.

21 Mar 2022