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).

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

Horizon Discovery to offer novel base editing technology for clinical research

Horizon Discovery today announced that it will provide access to a novel base editing technology licensed from Rutgers for exclusive use in therapeutic, diagnostic and services applications.

From Horizon Discovery 14 Jan 2020

Researchers find key to preventing muscular dystrophy-related heart disease

A Rutgers-led team may have found the key to preventing Duchenne muscular dystrophy (DMD)-related heart disease, the leading cause of death in patients living with the disease.

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

Nanoparticles used to deliver CRISPR gene editing tools into the cell

Researchers from Tufts University and the Chinese Academy of Sciences have developed a novel method to effectively deliver the CRISPR/Cas9 (clustered regularly interspaced short palindromic repeat (CRISPR) associated protein 9) gene editing tools into the liver for genetic studies.

15 Jul 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

New discovery could improve therapies for Duchenne muscular dystrophy

A new multi-institution study spearheaded by researchers at Florida State University and the University of California, Los Angeles suggests a tiny protein could play a major role in combating heart failure related to Duchenne muscular dystrophy (DMD), the most common lethal genetic disorder among children.

16 Jun 2019

Duchenne Muscular Dystrophy News and Research

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

New form of gene editing slows ALS progression in mice

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

Simple accelerometers improve diagnosis of Duchenne muscular dystrophy

New gene correction therapy for hereditary muscular disease among children

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

Horizon Discovery to offer novel base editing technology for clinical research

Researchers find key to preventing muscular dystrophy-related heart disease

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

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

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

Nanoparticles used to deliver CRISPR gene editing tools into the cell

New drug provides hope for patients with Duchenne muscular dystrophy

New discovery could improve therapies for Duchenne muscular dystrophy

Study raises the prospect of new therapies to delay muscle atrophy

Study discovers origin of neurofibromas that develop throughout the skin of NF1 patients

Novo Biosciences achieves major milestones in its new drug to treat heart disease and DMD

Researchers develop new method to boost efficiency of CRISPR gene editing in DMD

Clinical study shows tolerability and safety of investigational drug in boys with DMD

Phase 1 data reinforce safety profile of new drug for treating Duchenne muscular dystrophy

Why Primary Cells Are Important for In Vitro ADME-Tox Studies

Recognizing CDKL5 Deficiency Disorder

Isolating Single T-cells of Interest using an Optofluidic Platform

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