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.
MD-CARE Act Amendments become law

MD-CARE Act Amendments become law

Capping a two-year advocacy campaign led by Parent Project Muscular Dystrophy, today President Barack Obama signed the MD-CARE Act Amendments of 2014 into law, providing a significant victory to the Duchenne and broader muscular dystrophy communities. [More]
Research could help eventual treatment of degenerative muscle, brain diseases

Research could help eventual treatment of degenerative muscle, brain diseases

Our genetic information is stored in DNA, tiny strands of nucleic acid that contain instructions for the functioning of our bodies. To express this genetic data, our DNA is copied into RNA molecules, which then translate the instructions into proteins that perform tasks in our cells. [More]
Researchers examine why stem cells lose their capacity to repair damage as age increases

Researchers examine why stem cells lose their capacity to repair damage as age increases

As we age, stem cells throughout our bodies gradually lose their capacity to repair damage, even from normal wear and tear. [More]
Researchers develop novel technique to promote tissue repair in damaged muscles

Researchers develop novel technique to promote tissue repair in damaged muscles

Researchers at Sanford-Burnham Medical Research Institute (Sanford-Burnham) have developed a novel technique to promote tissue repair in damaged muscles. [More]

New approach to turn diseased cells into unique manufacturing sites for treating muscular dystrophy

In a new study that could ultimately lead to many new medicines, scientists from the Florida campus of The Scripps Research Institute (TSRI) have adapted a chemical approach to turn diseased cells into unique manufacturing sites for molecules that can treat a form of muscular dystrophy. [More]
Minnesota researchers develop animal research model for FSHD

Minnesota researchers develop animal research model for FSHD

Researchers at the University of Minnesota have developed an animal research model for facioscapulohumeral muscular dystrophy (FSHD) to be used for muscle regeneration research as well as studies of the effectiveness of potential therapies for FSHD. [More]
Research offers hope to patient suffering from Duchenne muscular dystrophy

Research offers hope to patient suffering from Duchenne muscular dystrophy

Research at Stockholm's KTH Royal Institute of Technology offers hope to those who suffer from Duchenne muscular dystrophy, an incurable, debilitating disease that cuts young lives short. [More]
Researchers restore missing repair protein in skeletal muscle of patients with muscular dystrophy

Researchers restore missing repair protein in skeletal muscle of patients with muscular dystrophy

Advances in the treatment of muscular dystrophy: For the first time, a research team has succeeded in restoring a missing repair protein in skeletal muscle of patients with muscular dystrophy. [More]
New gene therapy protects mice from life-threatening heart condition

New gene therapy protects mice from life-threatening heart condition

A new gene therapy developed by researchers at the University of Missouri School of Medicine has been shown to protect mice from a life-threatening heart condition caused by muscular dystrophy. [More]
Researchers use new gene editing method to correct mutation that leads to DMD

Researchers use new gene editing method to correct mutation that leads to DMD

UT Southwestern Medical Center researchers successfully used a new gene editing method to correct the mutation that leads to Duchenne muscular dystrophy (DMD) in a mouse model of the condition. [More]
Researchers find method to repair gene mutation causing autoimmune deficiency disease

Researchers find method to repair gene mutation causing autoimmune deficiency disease

Researchers at Karolinska Institutet in Sweden have found a method to repair the gene mutation causing agammaglobulinemia, an autoimmune deficiency disease that almost exclusively affects boys and in which the body lacks the ability to produce immunoglobulins (gamma globulin). [More]
Novogen receives funding support to commence studies in muscular dystrophy

Novogen receives funding support to commence studies in muscular dystrophy

The Australian biotechnology company, Novogen Limited today announced receipt of funding from the FSHD Global Research Foundation as part of the Company’s efforts to find effective treatments for a range of musculo-degenerative diseases including facioscapulohumeral dystrophy (FSHD). [More]
Scientists identify mechanism by which inherited neurological disease causes muscle weakness in men

Scientists identify mechanism by which inherited neurological disease causes muscle weakness in men

Researchers at University of California, San Diego School of Medicine have identified the mechanism by which a rare, inherited neurodegenerative disease causes often crippling muscle weakness in men, in addition to reduced fertility. [More]
Isoform: New protein offers novel therapeutic approach for patients with DMD

Isoform: New protein offers novel therapeutic approach for patients with DMD

Scientists have discovered a new form of dystrophin, a protein critical to normal muscle function, and identified the genetic mechanism responsible for its production. [More]
Small molecule may be able to convince damaged nerves to effectively rewire circuits

Small molecule may be able to convince damaged nerves to effectively rewire circuits

Frogs, dogs, whales, snails can all do it, but humans and primates can't. Regrow nerves after an injury, that is—while many animals have this ability, humans don't. [More]
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

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]