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.

Yale researchers identify possible treatment for Duchenne muscular dystrophy

Researchers at Yale have identified a possible treatment for Duchenne muscular dystrophy (DMD), a rare genetic disease for which there is currently no cure or treatment, by targeting an enzyme that had been considered "undruggable."

12 Sep 2020

Scientists solve decades old mystery of key molecule that fuels mitochondria

Penn Medicine researchers have solved a decades old mystery around a key molecule fueling the power plant of cells that could be exploited to find new ways to treat diseases, from neurodegenerative disorders to cancer.

10 Sep 2020

Researchers find link between dystrophic muscles and lymphatic system in mice with Duchenne disease

Researchers at the Universities of Maynooth and Bonn discover a new connection in muscular dystrophy Duchenne muscular dystrophy (DMD) is the most common muscle disease in children and is passed on by X-linked recessive inheritance.

27 Aug 2020

FDA approves new treatment option for patients with rare Duchenne muscular dystrophy mutation

Today, the U.S. Food and Drug Administration granted accelerated approval to Viltepso (viltolarsen) injection for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping.

12 Aug 2020

Heart cell treatment can benefit patients, shows study

More than three years after a clinical trial was prematurely ended for failing to show progress in healing heart attack scars, a prominent peer-reviewed journal is publishing some surprising results showing that the heart cell treatment does benefit patients.

4 Aug 2020

C-Path gets positive response from FDA for drug-induced skeletal muscle injury safety biomarkers

Critical Path Institute announced today that the Biomarker Qualification Program at the Center for Drug Evaluation and Research in the U.S. Food and Drug Administration issued a positive response to the Letter of Intent developed by C-Path's Predictive Safety Testing Consortium and Duchenne Regulatory Science Consortium, for a panel of four safety biomarkers of acute drug-induced skeletal muscle injury.

23 Jul 2020

Erectile dysfunction drugs lower the accumulation of toxic proteins in zebrafish models

The aberrant buildup of misfolded proteins is a hallmark of a host of disorders, including neurodegenerative diseases such as Alzheimer's and Parkinson's.

22 Jul 2020

Scientific evidence shows GLDH is a safe biomarker for drug-induced liver injury

Critical Path Institute (C-Path) announced today that the Biomarker Qualification Program (BQP) at the Center for Drug Evaluation and Research (CDER) from the U.S. Food and Drug Administration (FDA) issued a positive response to the Qualification Plan (QP) for glutamate dehydrogenase (GLDH) as a safety biomarker for drug-induced liver injury (DILI), developed by C-Path's Predictive Safety Testing Consortium (PSTC) and Duchenne Regulatory Science Consortium (D-RSC).

22 Jul 2020

Researchers use gene-editing techniques to visualize protein action in multicellular animals

Duke University researchers have made the first time-lapse movies of the sheet-like latticework that surrounds and supports most animal tissues.

9 Jul 2020

Experimental drug for rare ALS shows promise in early clinical trial

An experimental drug for a rare, inherited form of amyotrophic lateral sclerosis (ALS) has shown promise in a phase 1/phase 2 clinical trial conducted at Washington University School of Medicine in St. Louis, Massachusetts General Hospital in Boston and other sites around the world and sponsored by the pharmaceutical company Biogen Inc.

8 Jul 2020

New editorial published following adverse events of gene therapy clinical trial

The Editor-in-Chief of Human Gene Therapy, the first journal devoted to the field of gene therapy, and one of the world's leading experts on gene therapy have co-authored a new editorial, Moving Forward After Two Deaths in a Gene Therapy Trial of Myotubular Myopathy, in response to the news of two deaths in a now-halted gene therapy clinical trial.

3 Jul 2020

Researchers develop potential new therapy for common form of muscular dystrophy

Researchers have designed a potential new treatment for one of the most common forms of muscular dystrophy, according to a new study published today in the Proceedings of the National Academy of Sciences.

29 Jun 2020

Micro-dystrophin gene therapy can improve outcomes in children with Duchenne muscular dystrophy

Researchers from Nationwide Children's Hospital have published in JAMA Neurology results from the first four patients treated in the first clinical trial of systemic delivery of micro-dystrophin gene therapy in children with Duchenne muscular dystrophy (DMD) - and initial findings suggest that the therapy can provide functional improvement that is greater than that observed under the standard of care.

16 Jun 2020

Antisense Therapeutics: Poster presentation on the Muscular Dystrophy Association Virtual Conference 2020 website

Antisense Therapeutics, an Australian biopharmaceutical company developing and commercializing antisense pharmaceuticals for unmet need in rare diseases announces a virtual poster presentation on the Muscular Dystrophy Association Virtual 2020 Conference website.

15 Jun 2020

Does COVID-19 injure the placenta of pregnant women?

News-Medical speaks to Dr. Jeffery Goldstein about whether COVID-19 is damaging the placentas of pregnant women and what women can do to protect themselves.

12 Jun 2020

‘Why do we always get hit first?’ Proposed budget cuts target vulnerable Californians

Shirley Madden, 83, relies on a caregiver and her two grown daughters to remain living at home — and not in a nursing home.

1 Jun 2020

Using gene therapy to help the body to make its own cancer drugs

Imagine that we could instruct our bodies to make the drugs they need themselves. The gene technology that makes this possible is called mRNA therapy, which may become a major tool in the treatment of multiple diseases.

29 May 2020

Study provides groundwork for restoring nerve-muscle connection in ALS

Patients with amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease, lose muscle control as nerve cells or neurons in the brain and spinal cord degenerate and can no longer send signals to muscles.

28 May 2020

UTSW/Children's Health joint program approved as Certified Duchenne Care Center

A joint program of UT Southwestern Medical Center and Children's Health has been approved as a Certified Duchenne Care Center by Parent Project Muscular Dystrophy, the nation's most comprehensive nonprofit organization focused on finding a cure for Duchenne muscular dystrophy.

27 May 2020

Scientists identify key cell signaling pathway that drives muscle loss

Researchers at the University of Nebraska Medical Center have identified a key cell signaling pathway that drives the devastating muscle loss, or cachexia, suffered by many cancer patients.

22 May 2020