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).

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

MDA celebrates FDA approval of new spinal muscular atrophy drug

The Muscular Dystrophy Association today celebrated news of the U.S. Food and Drug Administration's decision to grant approval for nusinersen (brand name Spinraza), the first disease-modifying drug to treat the most common genetic cause of death in infants.

23 Dec 2016

Cedars-Sinai receives $7.3 million grant to test safety of novel cell-based therapy in treating PAH

Researchers from the Cedars-Sinai Heart Institute and the Cedars-Sinai Department of Medicine are expanding their ongoing evaluation of a novel cell-based therapeutic candidate into the area of pulmonary arterial hypertension (PAH).

6 Dec 2016

New drug treatment can override genetic fault that causes choroideremia

Researchers with funding from Fight for Sight have demonstrated that a new drug treatment for cystic fibrosis and Duchenne muscular dystrophy can override a genetic fault that causes choroideremia – a severe blinding disorder.

5 Dec 2016

FDA approval of controversial drug for Duchenne muscular dystrophy raises concern and hope

In September, the Food and Drug Administration approved Exondys, a controversial treatment for Duchenne muscular dystrophy based on tenuous data from just 12 patients.

17 Nov 2016

Fight for Sight, Thomas Pocklington collaborate to fund new neuro-ophthalmology research

Retinal activity in autism, ADHD and Duchenne muscular dystrophy

16 Nov 2016

Immune system plays important role in Duchenne muscular dystrophy, research reveals

A new paper, co-written by faculty at Binghamton University, State University of New York, increases the understanding of Duchenne muscular dystrophy (DMD)—one of the most common lethal genetic disorders—and points to potential therapeutic approaches.

14 Oct 2016

First comprehensive center opened in Tri-State area to mark new era in care of DMD patients

Nearly 1,000 boys in the New York Tri-State area have been diagnosed with Duchenne Muscular Dystrophy (DMD) and, until now, had to travel out of the state for comprehensive care.

7 Oct 2016