Muscular Dystrophy News and Research RSS Feed - Muscular Dystrophy News and Research

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

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). [More]
New drug treatment can override genetic fault that causes choroideremia

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. [More]
Research provides new insight into how diseases that disconnect brain and body occur

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. [More]
ASGCT seeks to educate public and policy-makers on fundamentals of gene editing

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. [More]
FDA approval of controversial drug for Duchenne muscular dystrophy raises concern and hope

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. [More]
Fight for Sight, Thomas Pocklington collaborate to fund new neuro-ophthalmology research

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

Retinal activity in autism, ADHD and Duchenne muscular dystrophy [More]
Home non-invasive ventilation for COPD: an interview with Dr Holger Woehrle

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. [More]
Immune system plays important role in Duchenne muscular dystrophy, research reveals

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

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. [More]
Department of Defense funds cardiac cell therapy trial for heart failure patients

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. [More]
CUMC researchers uncover new details of intracellular channel that controls skeletal muscle

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. [More]
Sarepta Therapeutics wins accelerated approval from FDA for Duchenne muscular dystrophy drug

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). [More]
New research could pave way for safe, effective treatment for muscular dystrophy

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. [More]
Christopher & Dana Reeve Foundation awards Quality of Life grants to 79 nonprofit organizations

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. [More]
MDI Biological Laboratory researchers receive patent for novel heart disease drug

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. [More]
Penn study sheds light on role of essential compound in maintaining optimal muscle function

Penn study sheds light on role of essential compound in maintaining optimal muscle function

Maintaining proper levels of an essential helper molecule is crucial for optimal muscle function, according to a study led by Joseph Baur, PhD, an assistant professor of Physiology in the Perelman School of Medicine at the University of Pennsylvania. [More]
New gene therapeutic approach could save people suffering from muscle wasting disease

New gene therapeutic approach could save people suffering from muscle wasting disease

A discovery by Washington State University scientist Dan Rodgers and collaborator Paul Gregorevic could save millions of people suffering from muscle wasting disease. [More]
Key gene controls ability of adult stem cells to regenerate muscle after injury, study finds

Key gene controls ability of adult stem cells to regenerate muscle after injury, study finds

A key gene enables the repair of injured muscle throughout life. This is the finding of a study in mice led by researchers at NYU Langone Medical Center and the University of Colorado at Boulder, and published online July 21 in Cell Reports. [More]
Protein present on stem cell surface can boost growth of damaged muscle tissue

Protein present on stem cell surface can boost growth of damaged muscle tissue

Johns Hopkins University biologists have found that a protein that plays a key role in the lives of stem cells can bolster the growth of damaged muscle tissue, a step that could potentially contribute to treatments for muscle degeneration caused by old age and diseases such as muscular dystrophy. [More]
Scientists explore black box of genome biology

Scientists explore black box of genome biology

Scientists at Florida State University, Baylor College of Medicine and the Broad Institute of Harvard and MIT have broken ground in a little-understood area of human genetics. [More]
Advertisement
Advertisement