Muscular Dystrophy News and Research

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The muscular dystrophies (MD) are a group of more than 30 genetic diseases characterized by progressive weakness and degeneration of the skeletal muscles that control movement. Some forms of MD are seen in infancy or childhood, while others may not appear until middle age or later. The disorders differ in terms of the distribution and extent of muscle weakness (some forms of MD also affect cardiac muscle), age of onset, rate of progression, and pattern of inheritance.

Study reports volume and cost of in-home care for children with special medical conditions

U.S. families provide nearly $36 billion annually in uncompensated medical care at home to children who have special health care needs, such as muscular dystrophy and cystic fibrosis, according to a large national study.

28 Dec 2016

Study tracks parents' unpaid time assisting children with special health care needs

About half of U.S. children with special health care needs -- 5.6 million children -- receive medical care from uncompensated family members worth billions of dollars, finds a large national study led by Boston Children's Hospital and the University of Southern California (USC).

27 Dec 2016

Ionis announces FDA approval of first SMA drug in the U.S for pediatric and adult patients

Ionis Pharmaceuticals, Inc. announced today that the U.S. Food and Drug Administration has approved SPINRAZATM (nusinersen) under Priority Review for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients.

26 Dec 2016

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

TSRI scientists develop new methods to design precision medicines against disease-causing RNAs

Scientists on the Florida campus of The Scripps Research Institute have developed broad methods to design precision medicines against currently incurable diseases caused by RNA.

20 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

Research provides new insight into how diseases that disconnect brain and body occur

A huge colony of receptors must be optimally positioned and functioning on our muscle cells for our brains to talk with our bodies so we can walk and breathe.

29 Nov 2016

ASGCT seeks to educate public and policy-makers on fundamentals of gene editing

On Nov. 21, 2016, the American Society of Gene & Cell Therapy released Therapeutic Gene Editing: an ASGCT White Paper, intended to provide policy-makers, patient advocates, and the interested public with the necessary background information in anticipation of an upcoming consensus report on human gene editing from the National Academy of Sciences and National Academy of Medicine (NAS), expected to be released in early 2017.

24 Nov 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

Home non-invasive ventilation for COPD: an interview with Dr Holger Woehrle

The key symptom of COPD is breathlessness, especially during daily activity, and the breathlessness is in addition to cough and chest tightness. The difficulty in managing this disease is that it's a disease that progresses with age.

From ResMed Corp 18 Oct 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

Department of Defense funds cardiac cell therapy trial for heart failure patients

Building on the results of a recent Cedars-Sinai Heart Institute study published just six months ago, the Department of Defense has awarded a $10 million grant to fund a cardiac cell therapy trial for patients diagnosed with a common but difficult-to-treat form of heart failure.

28 Sep 2016

CUMC researchers uncover new details of intracellular channel that controls skeletal muscle

Using high-resolution electron microscopy, Columbia University Medical Center researchers have uncovered new details of the structure and function of an intracellular channel that controls the contraction of skeletal muscle.

22 Sep 2016

Sarepta Therapeutics wins accelerated approval from FDA for Duchenne muscular dystrophy drug

The U.S. Food and Drug Administration today approved Exondys 51 (eteplirsen) injection, the first drug approved to treat patients with Duchenne muscular dystrophy (DMD).

19 Sep 2016

New research could pave way for safe, effective treatment for muscular dystrophy

New research has shown that the corticosteroid deflazacort is a safe and effective treatment for Duchenne muscular dystrophy. The findings, which appear this month in the journal Neurology, could pave the way for first U.S.-approved treatment for the disease.

9 Sep 2016

Christopher & Dana Reeve Foundation awards Quality of Life grants to 79 nonprofit organizations

The Christopher & Dana Reeve Foundation, a national nonprofit dedicated to improving quality of life for individuals living with paralysis, announced it has awarded $575,704 in Quality of Life grants to 79 nonprofit organizations nationwide.

27 Aug 2016

MDI Biological Laboratory researchers receive patent for novel heart disease drug

The U.S. Patent and Trademark Office has announced that it will grant a patent to MDI Biological Laboratory scientists Voot P. Yin, Ph.D., and Kevin Strange, Ph.D., and their collaborator Michael Zasloff, M.D., Ph.D., for use of the small molecule MSI-1436 to stimulate the repair and regeneration of heart tissue damaged by injuries such as a heart attack.

23 Aug 2016