Duchenne Muscular Dystrophy News and Research

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Duchenne muscular dystrophy (DMD) is a progressive muscle disorder that causes the loss of both muscle function and independence. DMD is perhaps the most prevalent of the muscular dystrophies and is the most common lethal genetic disorder diagnosed during childhood today. Each year, approximately 20,000 children worldwide are born with DMD (one of every 3,500 male children).

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

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

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

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

Reactivating Piezo1 protein could help repair broken down, dystrophic muscles

One protein, Piezo1, is key to marshalling muscle stem cells' unique shapes and response to injuries, but it is in low supply in those with Duchenne muscular dystrophy, according to a team at the Perelman School of Medicine at the University of Pennsylvania.

18 Mar 2022

Cellular therapy offers promise for patients with Duchenne muscular dystrophy, trial shows

A clinical trial at UC Davis Health and six other sites showed that a cellular therapy offers promise for patients with late-stage Duchenne muscular dystrophy (DMD), a rare genetic disorder causing muscle loss and physical impairments in young people.

10 Mar 2022

Glucocorticoid steroids improve muscle performance through distinct, sex-specific molecular pathways

Glucocorticoid steroids improved muscle performance through distinct, sex-specific molecular mechanisms in mice, according to a Northwestern Medicine study published in the Journal of Clinical Investigation.

7 Mar 2022

Inside the tactical tug of war over the controversial Alzheimer’s drug

The drug industry, patient advocates, and congressional Republicans have all attacked federal officials' decision to decline routine Medicare coverage for a controversial Alzheimer's drug.

16 Feb 2022

Study reveals link between muscular dystrophy and sphingolipids

In a new study, the group of Johan Auwerx at EPFL's School of Life Sciences has made the first connection between muscular dystrophy and sphingolipids, a group of bioactive lipids. The study is published in Science Advances.

29 Jan 2022

Novel gene therapy for diseased muscle fiber repair

In this interview, News-Medical speaks to Dr. Jyoti K. Jaiswal about his latest research into gene therapies for limb-girdle muscular dystrophy 2B.

24 Jan 2022

Single-patient pilot study shows the ability of antisense oligonucleotide to suppress mutant ALS gene

Using a short, synthetic chain of chemically modified nucleotides engineered in the RNA Therapeutics Institute at UMass Chan Medical School, Robert H. Brown Jr., DPhil, MD, Jonathan Watts, PhD, and colleagues have shown the ability to suppress mutant forms of an ALS gene known as C9ORF72 in a single-patient pilot study.

24 Dec 2021

New consensus-based recommendations designed to improve quality of life for DMD patients

Many more adults with Duchenne muscular dystrophy (DMD) are living longer thanks to improvements in treatment, however international standards of care have not yet fully addressed the complex needs of these patients.

9 Dec 2021

First pediatric-only guidelines for the use of pacemakers and implantable defibrillators

Children's Hospital Los Angeles cardiologist Michael Silka, MD, helped to pioneer the development of indications for the use of pacemakers and implantable defibrillators in young patients.

6 Dec 2021