Dystrophin News and Research

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Dystrophin is a rod-shaped cytoplasmic protein, and a vital part of a protein complex that connects the cytoskeleton of a muscle fiber to the surrounding extracellular matrix through the cell membrane.

Study unlocks some muscle cell secrets

A muscle fiber consists of just one cell, but many nuclei. A team at the MDC led by Professor Carmen Birchmeier has now shown just how varied these nuclei are.

11 Dec 2020

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

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

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

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

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

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

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

New study indicates novel therapies for Duchenne muscular dystrophy

When an actor is unable to perform in the theatre, an understudy--ideally one with some practice in the role--can take her place on stage.

25 Apr 2020

Model that sheds light on severe Duchenne muscular dystrophy could pave the way for new therapies

Researchers have developed an experimental model of severe Duchenne muscular dystrophy that offers insight into the disease.

26 Mar 2020

Small molecules may open the door to new therapies for Duchenne muscular dystrophy

Researchers identified a group of small molecules that may open the door to developing new therapies for Duchenne muscular dystrophy, an as-yet-uncured disease that results in devastating muscle weakening and loss.

24 Feb 2020

Study reveals molecular pathogenesis of muscular dystrophy-associated cardiomyopathy

In a study recently published in Nature Communications scientists at Okayama University describe the detailed molecular pathogenesis of muscular dystrophy-associated cardiomyopathy in mice lacking the fukutin gene (Fktn), the causative gene for Fukuyama muscular dystrophy.

13 Feb 2020

New gene correction therapy for hereditary muscular disease among children

Duchenne type muscular dystrophy is the most common hereditary muscular disease among children, leaving them wheelchair-bound before the age of twelve and reducing life expectancy.

27 Jan 2020

PerkinElmer provides newborn screening assay for PPMD’s New York State pilot program

PerkinElmer, Inc., a global leader committed to innovating for a healthier world, today announced that it is providing the newborn screening assay for Parent Project Muscular Dystrophy’s Newborn Screening Pilot for Duchenne Muscular Dystrophy (Duchenne).

From PerkinElmer 14 Jan 2020

Experimental treatment shows early promise to help nearly half of DMD patients

A new therapeutic being tested by University of Alberta researchers is showing early promise as a more effective treatment that could help nearly half of patients with Duchenne muscular dystrophy.

25 Sep 2019

Cheaper drug equally effective in preserving cardiovascular function in boys with DMD

A new clinical trial conducted at The Ohio State University Wexner Medical Center found a cost-effective generic medication works just as well as a more expensive drug in preserving cardiovascular function in boys with Duchenne muscular dystrophy (DMD).

24 Sep 2019

Study: Gene editing may correct genetic mutation responsible for Duchenne muscular dystrophy

Duchenne muscular dystrophy (DMD) is a rare but devastating genetic disorder that causes muscle loss and physical impairments.

18 Sep 2019

New drug provides hope for patients with Duchenne muscular dystrophy

The results from three clinical trials have shown that a new drug can successfully delay the progression of Duchenne muscular dystrophy.

10 Jul 2019