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).

SLU scientists provide promising approach in designing new drugs for DMD

In a paper published in the Nature journal Scientific Reports, Saint Louis University researchers report that a new drug reduces fibrosis (scarring) and prevents loss of muscle function in an animal model of Duchenne muscular dystrophy (DMD), providing a promising approach in designing new medications for those suffering from DMD.

8 Dec 2017

UT Southwestern researchers develop improved gene-editing technique to correct DMD mutations

Regenerative medicine researchers at UT Southwestern Medical Center developed an improved and simplified gene-editing technique using CRISPR/Cas9 tools to correct a common mutation that causes Duchenne muscular dystrophy.

30 Nov 2017

New initiative aims to improve chances of medicines receiving positive HTA decisions

A unique initiative launched today by muscular dystrophy charity Duchenne UK is set to radically simplify the way necessary evidence is generated for submissions made to health technology appraisals bodies such as the National Institute for Health and Care Excellence and the Haute Autorité de Santé.

22 Nov 2017

Clinical trial results could lead to treatment of heart disease in Duchenne muscular dystrophy patients

After boys and young men with Duchenne muscular dystrophy received cardiac progenitor cell infusions, medical tests indicated that the patients' hearts appeared improved, results from a new study show. Patients in the study also scored higher on arm strength tests after receiving the cell infusions.

15 Nov 2017

Study identifies predictors of poor outcomes in Duchenne muscular dystrophy patients

A new study from UT Southwestern suggests that more people with Duchenne muscular dystrophy could live longer by identifying and more aggressively treating patients with certain risk factors.

20 Oct 2017

CRISPR-Gold corrects gene mutations in mice with Duchenne muscular dystrophy

Scientists at the University of California, Berkeley, have engineered a new way to deliver CRISPR-Cas9 gene-editing technology inside cells and have demonstrated in mice that the technology can repair the mutation that causes Duchenne muscular dystrophy, a severe muscle-wasting disease.

4 Oct 2017

University Of Minnesota scientists describe breakthrough in research related to FSHD

A new study published in the journal Nature Communications describes a breakthrough in research related to facioscapulohumeral muscular dystrophy (FSHD). The debilitating genetic disease - which has no approved treatment - affects an estimated 38,000 Americans and causes degeneration and wasting of the skeletal muscles.

15 Sep 2017

Penn researchers find abnormally shortened telomeres in young DMD patients’ muscle stem cells

Researchers from the Perelman School of Medicine at the University of Pennsylvania have made a discovery about muscular dystrophy disorders that suggest new possibilities for treatment.

7 Sep 2017

Cardiac stem cells from young hearts can make older hearts young, study says

A new study from the Cedars-Sinai Heart Institute, Los Angeles, published by the European Heart Journal, forecasts the use of infusions of cardiac stem cells obtained from young hearts in reversing the process of aging in the human heart.

16 Aug 2017

Cardiac stem cell infusions could help reverse aging process in the heart, study shows

Cardiac stem cell infusions could someday help reverse the aging process in the human heart, making older ones behave younger, according to a new study from the Cedars-Sinai Heart Institute.

14 Aug 2017

Expert highlights CRISPR’s applications, limitations, and ethical concerns

"Good morning, doctor, I am here for my gene editing appointment." In the future, could this be a greeting heard in physician offices around the world? With the introduction of CRISPR technology, genetic material can now be more easily and precisely edited, even creating changes that can subsequently be inherited by offspring.

1 Aug 2017

Preclinical study demonstrates efficacy of gene therapy in treatment of Duchenne muscular dystrophy

Researchers from Genethon, the AFM-Telethon laboratory, Inserm (UMR 1089, Nantes) and the University of London (Royal Holloway) demonstrated the efficacy of an innovative gene therapy in the treatment of Duchenne muscular dystrophy.

25 Jul 2017

Cellular antennae play key role in muscle-to-fat transformation

Like it or not, as we age, our muscle cells are slowly exchanged, one by one, for fat cells. This process quickens when we injure a muscle, and an extreme form of this process is also seen in muscle-wasting diseases such as Duchenne muscular dystrophy (DMD).

13 Jul 2017

Weekly doses of steroids help hasten recovery in muscle injuries

Weekly doses of glucocorticoid steroids, such as prednisone, help speed recovery in muscle injuries, reports a new Northwestern Medicine study. The weekly steroids also repaired muscles damaged by muscular dystrophy.

17 May 2017

Preclinical data reinforces potential efficacy, durability of investigational gene therapy for DMD

Solid Biosciences announced today that new data from two preclinical studies reinforce the potential of its investigational microdystrophin gene therapy, SGT-001, to be an effective treatment approach for Duchenne muscular dystrophy.

11 May 2017

Actress Kiruna Stamell argues about gene editing with Dr Christopher Gyngell

Two papers published today by the Journal of the Royal Society of Medicine, debate gene editing and the health of future generations.

29 Apr 2017

Researchers correct Duchenne muscular dystrophy using gene-editing alternative

Using the new gene-editing enzyme CRISPR-Cpf1, researchers at UT Southwestern Medical Center have successfully corrected Duchenne muscular dystrophy in human cells and mice in the lab.

13 Apr 2017

Early treatment with heart failure drug can improve cardiac function in young boys with DMD

Researchers at The Ohio State University Ross Heart Hospital and Nationwide Children's Hospital have shown early treatment with the heart failure medication eplerenone can improve heart function in young boys with Duchenne muscular dystrophy (DMD) and stabilize heart function in older boys with the disease.

7 Mar 2017

FDA approves new treatment for wide range of patients with Duchenne muscular dystrophy

The U.S. Food and Drug Administration today approved Emflaza (deflazacort) tablets and oral suspension to treat patients age 5 years and older with Duchenne muscular dystrophy (DMD), a rare genetic disorder that causes progressive muscle deterioration and weakness.

9 Feb 2017

Researchers find striking differences between countries, age groups in access to proper care for DMD

Duchenne muscular dystrophy (DMD), a progressive muscle disease affecting one in 3800-6300 live male births and leads to ambulatory loss, respiratory problems, cardiomyopathy, and early death of patients in their 20s or 30s.

4 Jan 2017