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.
UTHealth scientists identify inhibitory switch to prevent peripheral vascular disease

UTHealth scientists identify inhibitory switch to prevent peripheral vascular disease

Millions of people in the United States have a circulatory problem of the legs called peripheral vascular disease. It can be painful and may even require surgery in serious cases. This disease can lead to severe skeletal muscle wasting and, in turn, limb amputation. [More]
Splice-switching oligonucleotide drugs alter editing of gene transcript

Splice-switching oligonucleotide drugs alter editing of gene transcript

In splice-switching, an innovative therapeutic approach, targeted oligonucleotide drugs alter the editing of a gene transcript to produce the desired form of a protein. [More]
PTC Therapeutics initiates reimbursed expanded access program for Translarna

PTC Therapeutics initiates reimbursed expanded access program for Translarna

PTC Therapeutics, Inc. today announced the initiation of a reimbursed expanded access program (EAP). PTC's EAP program is intended to make Translarna (ataluren) available to patients before commercial availability in certain countries. [More]
FDA grants Fast Track designation to Akashi Therapeutics' HT-100 for treatment of DMD

FDA grants Fast Track designation to Akashi Therapeutics' HT-100 for treatment of DMD

Akashi Therapeutics, Inc., announced today that the U.S. Food and Drug Administration has granted Fast Track designation to the company's most advanced product candidate, HT-100 (delayed-release halofuginone), an orally available, small molecule drug candidate intended to reduce fibrosis and inflammation and promote healthy muscle regeneration in boys with DMD. [More]
Isis Pharmaceuticals begins ISIS-DMPK Rx Phase 1 study for treatment of Myotonic Dystrophy Type 1

Isis Pharmaceuticals begins ISIS-DMPK Rx Phase 1 study for treatment of Myotonic Dystrophy Type 1

Isis Pharmaceuticals, Inc. announced today that it has initiated a Phase 1 study for ISIS-DMPKRx. Isis earned a $14 million milestone payment from Biogen Idec associated with this achievement. [More]
New gene involved in Parkinson's disease, find researchers

New gene involved in Parkinson's disease, find researchers

A team of UCLA researchers has identified a new gene involved in Parkinson's disease, a finding that may one day provide a target for a new drug to prevent and potentially even cure the debilitating neurological disorder. [More]
Nationwide Insurance Foundation donates $10M to Nationwide Children's Hospital

Nationwide Insurance Foundation donates $10M to Nationwide Children's Hospital

Nationwide Children's Hospital today announced that it has received a $10 million gift from the Nationwide Insurance Foundation. [More]
EMA's CHMP gives positive opinion for PTC Therapeutics' Translarna

EMA's CHMP gives positive opinion for PTC Therapeutics' Translarna

PTC Therapeutics, Inc. today announced that following its request for re-examination, the Committee for Medicinal Products for Human Use of the European Medicines Agency has adopted a positive opinion regarding the company's application for a conditional marketing authorization of Translarna (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) in ambulatory patients aged five years and older. [More]
DNA-directed RNA interference and hepatitis C: an interview with Carl Stubbings, Chief Business Officer, Benitec Biopharma

DNA-directed RNA interference and hepatitis C: an interview with Carl Stubbings, Chief Business Officer, Benitec Biopharma

RNA interference (RNAi) is a naturally occurring process, which cells can use to silence, or ‘turn off’ unwanted genes. [More]
Researchers identify heart-specific form of protein that protects against irregular heartbeats

Researchers identify heart-specific form of protein that protects against irregular heartbeats

Researchers at the Cedars-Sinai Heart Institute have identified a heart-specific form of a protein, BIN1, responsible for sculpting tiny folds in pockets that are present on the surface of heart muscle cells. [More]
Eli and Edythe Broad Foundation gift $4 million to support UCLA research

Eli and Edythe Broad Foundation gift $4 million to support UCLA research

Two new gifts from The Eli and Edythe Broad Foundation to UCLA totaling $4 million will fund research in stem cell science and digestive diseases and support the recruitment of key faculty at two renowned research centers. [More]
Viagra may help boys suffering from muscular dystrophy disease

Viagra may help boys suffering from muscular dystrophy disease

Drugs used to treat erectile dysfunction may be able to slow disease progression in boys who suffer from the muscle wasting disease Duchenne muscular dystrophy. [More]
Commonly used drug restores blood flow to oxygen-starved muscles of boys with muscular dystrophy

Commonly used drug restores blood flow to oxygen-starved muscles of boys with muscular dystrophy

Cedars-Sinai Heart Institute researchers have found that a commonly prescribed drug restores blood flow to oxygen-starved muscles of boys with Duchenne muscular dystrophy, a genetic muscle-wasting disease that rarely is seen in girls but affects one in 3,500 male babies, profoundly shortening life expectancy. It is the most common fatal disease that affects children. [More]
Common drug may improve blood flow in muscles of boys with Duchenne muscular dystrophy

Common drug may improve blood flow in muscles of boys with Duchenne muscular dystrophy

A drug typically prescribed for erectile dysfunction or increased pressure in the arteries may help improve blood flow in the muscles of boys with Duchenne muscular dystrophy, according to a study published in the May 7, 2014, online issue of Neurology®, the medical journal of the American Academy of Neurology. [More]
Novogen partners with Genea Biocells to accelerate testing for neurodegenerative diseases

Novogen partners with Genea Biocells to accelerate testing for neurodegenerative diseases

Novogen Limited, an oncology drug development company, today announced a collaboration with Australian company, Genea Biocells, to accelerate testing of their super-benzopyran (SBP) drugs for degenerative diseases of the nervous system and muscles. [More]
Positive results from pivotal clinical trial of Trophos’ olesoxime in SMA patients to be presented at AAN 2014

Positive results from pivotal clinical trial of Trophos’ olesoxime in SMA patients to be presented at AAN 2014

Trophos and AFM-Telethon today announce that data from the pivotal clinical trial of Trophos’ lead product candidate olesoxime in spinal muscular atrophy (SMA) will be presented during the 66th American Academy of Neurology annual meeting to be held in Philadelphia, PA, USA, from April 26 to May 3 2014. [More]
Researchers use RNA sequencing to look at early- and late-stage development of the heart

Researchers use RNA sequencing to look at early- and late-stage development of the heart

Most people think the development of the heart only happens in the womb, however the days and weeks following birth are full of cellular changes that play a role in the structure and function of the heart. Using mouse models, researchers at Baylor College of Medicine have now been able to categorize the alternative splicing (the process in which genes code proteins, determining their role) that takes place during these changes and what mechanisms they affect. [More]
Research findings can impact lives of people with autism today

Research findings can impact lives of people with autism today

A new study from investigators with the Autism Genome Project, the world's largest research project on identifying genes associated with risk for autism, has found that the comprehensive use of copy number variant (CNV) genetic testing offers an important tool in individualized diagnosis and treatment of autism. [More]

Race to Yes campaign lauds Sarepta's plans to seek FDA approval for Duchenne treatment

The Race to Yes campaign today lauded drug maker Sarepta on its plans to move forward immediately to seek FDA approval of the first drug to successfully treat Duchenne muscular dystrophy, the world's leading genetic killer of children. [More]
Sarepta Therapeutics plans to submit eteplirsen NDA for treatment of Duchenne muscular dystrophy

Sarepta Therapeutics plans to submit eteplirsen NDA for treatment of Duchenne muscular dystrophy

Sarepta Therapeutics, Inc., a developer of innovative RNA-based therapeutics, today announced it plans to submit a New Drug Application (NDA) to the U.S. Food and Drug Administration by the end of 2014 for the approval of eteplirsen for the treatment of Duchenne muscular dystrophy (DMD). [More]