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).

Nationwide Children's Hospital pioneers new gene therapy for Duchenne muscular dystrophy

In a landmark moment for the Abigail Wexner Research Institute at Nationwide Children's, a 5-year-old from Bellefontaine, Ohio, received the first dose of a recently approved gene therapy for Duchenne muscular dystrophy at Nationwide Children's Hospital, where the therapy was invented and initially tested.

8 Aug 2023

New porcine model could help develop treatments for Dduchenne muscular dystrophy

The group of LMU scientist Eckhard Wolf has developed a porcine DMD model with a mutation, which mimics the hallmarks of the human disease but develops them in an accelerated mode.

12 Jul 2023

Alternative splicing in neurodegenerative disease and the promise of RNA-based therapeutic strategies

Alternative splicing, a clever way a cell generates many different variations of messenger RNAs -; single-stranded RNAs involved in protein synthesis -; and proteins from the same stretch of DNA, plays an important role in molecular biology.

21 Jun 2023

Manipulating a signaling protein to slow down muscular dystrophy

A team of researchers at the University of Houston College of Pharmacy is reporting that by manipulating TAK1, a signaling protein that plays an important role in development of the immune system, they can slow down disease progression and improve muscle function in Duchenne muscular dystrophy (DMD).

25 May 2023

Scarring to the collagen framework causes dysfunction in Duchenne muscular dystrophy

Muscles that ache after a hard workout usually don't hurt for long, thanks to stem cells that rush to the injured site along "collagen highways" within the muscle and repair the damaged tissue.

11 May 2023

Novel molecule may help overcome the devastating symptoms of Duchenne muscular dystrophy

In a new study published in The FASEB Journal, investigators demonstrated the potential of a molecule that may help overcome some of the devastating symptoms of Duchenne muscular dystrophy (DMD), the most common life-limiting congenital neuromuscular disorder.

5 Apr 2023

Rare Diseases Action Plan for England focuses on providing better care and reducing health inequalities

A new Rare Diseases Action Plan for England will be published today (Tuesday 28 February) that will ensure those living with these conditions receive better care and treatment, fairer access to testing and have continued support.

28 Feb 2023

Oxford-Harrington Rare Disease Centre hosts virtual event in honor of the upcoming Rare Disease Day

The Oxford-Harrington Rare Disease Centre, a partnership of the University of Oxford and Harrington Discovery Institute at University Hospitals, is hosting a virtual event on Monday, Feb. 27, 2023, in honor of the upcoming Rare Disease Day, which is celebrated across the globe on February 28 every year.

21 Feb 2023

Using motion capture technology and AI to monitor the progression of movement disorders

A multi-disciplinary team of researchers has developed a way to monitor the progression of movement disorders using motion capture technology and AI.

23 Jan 2023

Gene editing could offer a promising solution for treating patients after a heart attack

Editing a gene that prompts a cascade of damage after a heart attack appeared to reverse this inevitable course in mice, leaving their hearts remarkably unharmed, a new study by UT Southwestern scientists showed.

20 Jan 2023

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

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

Duchenne Muscular Dystrophy News and Research

Nationwide Children's Hospital pioneers new gene therapy for Duchenne muscular dystrophy

New porcine model could help develop treatments for Dduchenne muscular dystrophy

Alternative splicing in neurodegenerative disease and the promise of RNA-based therapeutic strategies

Manipulating a signaling protein to slow down muscular dystrophy

Scarring to the collagen framework causes dysfunction in Duchenne muscular dystrophy

Novel molecule may help overcome the devastating symptoms of Duchenne muscular dystrophy

Rare Diseases Action Plan for England focuses on providing better care and reducing health inequalities

Oxford-Harrington Rare Disease Centre hosts virtual event in honor of the upcoming Rare Disease Day

Using motion capture technology and AI to monitor the progression of movement disorders

Gene editing could offer a promising solution for treating patients after a heart attack

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

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

New discovery changes the understanding of Duchenne muscular dystrophy

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

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

Cancer drug helped delay progression of Duchenne muscular dystrophy in mice

Existing cancer drug shows promise for treating patients with muscular dystrophy

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

The promise of RNA medicine is targeted and personalized

Study supports daily steroid doses for children with Duchenne muscular dystrophy

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