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).

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

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

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

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

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

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

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

Temple researchers reverse muscle fibrosis associated with overuse injury in animals

Overuse injuries - think muscle strains, tennis elbow, and rotator cuff tears - are a considerable problem in the United States, especially among young athletes.

30 Mar 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

New form of gene editing slows ALS progression in mice

With a new CRISPR gene-editing methodology, scientists from the University of Illinois at Urbana-Champaign inactivated one of the genes responsible for an inherited form of amyotrophic lateral sclerosis - a debilitating and fatal neurological disease for which there is no cure.

26 Feb 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

Simple accelerometers improve diagnosis of Duchenne muscular dystrophy

Duchenne muscular dystrophy is the most common type of muscular dystrophy, affecting more than 10,000 males at birth per year in the United States with severe physical disability, chronic wasting and muscle deterioration.

11 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