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.

Genethon to start international clinical trial for treatment of Duchenne muscular dystrophy

Genethon, dedicated to designing and developing gene therapy products for rare diseases, received this Monday 30th of November the authorization from the ANSM, the French National Agency for Medicines and Health Products Safety, to start in France a multicentre international clinical trial for the treatment of Duchenne muscular dystrophy with product GNT 004.

2 Dec 2020

Novel ALS study effectively visualizes neuroinflammation

A novel ALS (amyotrophic lateral sclerosis) study has pooled data from multiple sites to effectively visualize neuroinflammation, which is key to developing drugs to treat the disease.

2 Dec 2020

New method could help determine the most appropriate treatment for HIV patients

A team co-led by a scientist at the University of California, Riverside, has developed a method to study how HIV mutates to escape the immune system in multiple individuals, which could inform HIV vaccine design.

30 Nov 2020

Adding exercise to genetic treatment reverses fatigue in mice with common myotonic dystrophy

Adding exercise to a genetic treatment for myotonic dystrophy type 1 (DM1) was more effective at reversing fatigue than administering the treatment alone in a study using a mouse model of the disease.

30 Nov 2020

Mother's protein found to be involved in genomic imprinting process

Mothers leave their mark on their children in many ways - and Australian researchers have discovered a protein called SMCHD1 is involved in this 'imprinting' process.

24 Nov 2020

CRISPR/Cas9 system is highly effective in treating metastatic cancers

Researchers at Tel Aviv University (TAU) have demonstrated that the CRISPR/Cas9 system is very effective in treating metastatic cancers, a significant step on the way to finding a cure for cancer.

19 Nov 2020

New strategy shows promises for treating RNA-repeat expansion disorders

A new strategy for treating a variety of diseases known as RNA-repeat expansion disorders, which affect millions of people, has shown promise in proof-of-principle tests conducted by scientists at Scripps Research.

8 Nov 2020

Genetic mutation may accelerate heart function decline in DMD patients

A mutation in the gene that causes cystic fibrosis may accelerate heart function decline in those with Duchenne muscular dystrophy (DMD), a new study by UT Southwestern researchers suggests.

4 Nov 2020

The best COVID warning system? Poop and pooled spit, says one Colorado school

Carol Wilusz's mornings now often start at 4 a.m., scanning the contents of undergraduates' feces. Specifically, scanning the data on how much coronavirus they flushed into the shadows, destined to be extracted from 17 manholes connected to dorm buildings on Colorado State University's Fort Collins campus.

3 Nov 2020

New study may help combat loss of muscle mass associated with aging

Researchers at the University of São Paulo (USP) in Brazil have discovered that the muscle regeneration promoted by aerobic exercise is mediated by changes in oxygen consumption by satellite cells, also known as skeletal muscle stem cells.

29 Oct 2020

Scientists identify new therapeutic targets for SARS-CoV-2

To identify new potential therapeutic targets for SARS-CoV-2, a team of scientists at the New York Genome Center, New York University, and the Icahn School of Medicine at Mount Sinai, performed a genome-scale, loss-of-function CRISPR screen to systematically knockout all genes in the human genome.

27 Oct 2020

UCLA researchers discover compound that stimulates muscle cells in mice

UCLA researchers have identified a compound that can reproduce the effect of exercise in muscle cells in mice. The findings are published in the journal Cell Reports Medicine.

21 Oct 2020

Leaking components from broken muscle fibers activate 'satellite' muscle stem cells

While building a muscle damage model in a cultured system, a research collaboration between Kumamoto University and Nagasaki University in Japan has found that components leaking from broken muscle fibers activate "satellite" muscle stem cells.

12 Oct 2020

Study elucidates the role of ERβ in growth of skeletal muscle in female mice

Researchers at Kumamoto University, Japan generated mice lacking the estrogen receptor beta (ERβ) gene, both fiber-specific and muscle stem cell-specific, which resulted in abnormalities in the growth and regeneration of skeletal muscle in female mice.

12 Oct 2020

Biodegradable microneedle patch for local therapeutic gene delivery

There is great potential in gene therapy for treating certain types of cancer and genetic defects, immunological diseases, wounds and infections. The therapies work by delivering genes into the patients' cells, which then produce therapeutic proteins to treat the affliction.

7 Oct 2020

NIH awards grants for using bioengineered models to improve clinical trial design

Clinical Trials on a Chip researchers plan to build and test common and rare disease models to help improve the clinical trial process.

30 Sep 2020

New drug offers hope for young boys with Duchenne muscular dystrophy

A new drug offers hope for young boys with the progressive neuromuscular disease Duchenne muscular dystrophy (DMD) by potentially offering an alternative to high-dose glucocorticoids that have significant side effects.

25 Sep 2020

Curi Bio Announces Mantarray™ Platform for Analysis of 3D Engineered Muscle Tissues for Discovery of New Therapeutics

Curi Bio today announced the Mantarray™ platform for human-relevant 3D engineered muscle tissue (EMT) analysis.

From Curi Bio 16 Sep 2020

Study uncovers a promising new therapeutic approach for Duchenne muscular dystrophy

Scientists at Sanford Burnham Prebys Medical Discovery Institute, Fondazione Santa Lucia IRCCS, and Università Cattolica del Sacro Cuore in Rome have shown that pharmacological (drug) correction of the content of extracellular vesicles released within dystrophic muscles can restore their ability to regenerate muscle and prevent muscle scarring (fibrosis).

15 Sep 2020

RCas9 gene therapy clears toxic RNA buildup in mouse model of myotonic dystrophy

Myotonic dystrophy type I is the most common type of adult-onset muscular dystrophy. People with the condition inherit repeated DNA segments that lead to the toxic buildup of repetitive RNA, the messenger that carries a gene's recipe to the cell's protein-making machinery.

14 Sep 2020