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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.
Isis Pharmaceuticals begins ISIS-DMPK Rx clinical study in DM1 patients

Isis Pharmaceuticals begins ISIS-DMPK Rx clinical study in DM1 patients

Isis Pharmaceuticals, Inc. announced today that it has initiated a study for ISIS-DMPK Rx in patients with Myotonic Dystrophy Type 1 (DM1). DM1 is a rare genetic neuromuscular disease caused by the production of toxic dystrophia myotonica-protein kinase (DMPK) RNA in cells. ISIS-DMPKRx is specifically designed to reduce toxic DMPK RNA. [More]
Cedars-Sinai Medical Center releases November tip sheet of story ideas

Cedars-Sinai Medical Center releases November tip sheet of story ideas

Following is the November 2014 tip sheet of story ideas from Cedars-Sinai Medical Center. [More]
Researchers show that iPS cells can be used to edit genetic mutations that cause DMD

Researchers show that iPS cells can be used to edit genetic mutations that cause DMD

Researchers at the Center for iPS Cell Research and Application, Kyoto University, show that induced pluripotent stem (iPS) cells can be used to correct genetic mutations that cause Duchenne muscular dystrophy (DMD). [More]
PPMD, Santhera Pharmaceuticals collaborate to study Duchenne muscular dystrophy

PPMD, Santhera Pharmaceuticals collaborate to study Duchenne muscular dystrophy

Parent Project Muscular Dystrophy, the leading advocacy organization working to end Duchenne muscular dystrophy (Duchenne) and Santhera Pharmaceuticals, a Swiss specialty pharmaceutical company focusing on the development and marketing of innovative pharmaceutical products for the treatment of mitochondrial and neuromuscular diseases, will collaborate on a benefit/risk study in Duchenne. [More]
Isis Pharmaceuticals announces initiation of ISIS-SMN Rx Phase 3 study in children with SMA

Isis Pharmaceuticals announces initiation of ISIS-SMN Rx Phase 3 study in children with SMA

Isis Pharmaceuticals, Inc. announced today the initiation of a pivotal Phase 3 study evaluating ISIS-SMNRx in approximately 120 non-ambulatory children with spinal muscular atrophy (SMA). [More]
Cardiac stem cell treatment restores heart function damaged by Duchenne muscular dystrophy

Cardiac stem cell treatment restores heart function damaged by Duchenne muscular dystrophy

Researchers at the Cedars-Sinai Heart Institute have found that injections of cardiac stem cells might help reverse heart damage caused by Duchenne muscular dystrophy, potentially resulting in a longer life expectancy for patients with the chronic muscle-wasting disease. [More]
A goodwill ambassador of palliative care

A goodwill ambassador of palliative care

Leanne Schoberl could have put anyone's picture on the home screen of her cell phone. "She loved American Idol winner Scotty McCreery. She envisioned marrying him one day," says Leanne's father John Schoberl. [More]
Murdoch University researchers receive NHMRC grant to develop genetic drugs for rare diseases

Murdoch University researchers receive NHMRC grant to develop genetic drugs for rare diseases

Murdoch University researchers from the Centre for Comparative Genomics have received almost $800,000 in funding from the National Health and Medical Research Council to develop genetic drugs to treat rare diseases. [More]
Mayo Clinic orthopedic surgeon explains how scoliosis affects Baby Boomers

Mayo Clinic orthopedic surgeon explains how scoliosis affects Baby Boomers

For many adults, the word scoliosis conjures up childhood memories of lining up in gym class for an examination by the school nurse. But scoliosis isn't just a pediatric condition. Curvature of the spine can develop in adults too, and the osteoporosis that can accompany menopause is a risk factor. [More]
Royal Holloway-led researchers to develop novel spinal cord injury treatment

Royal Holloway-led researchers to develop novel spinal cord injury treatment

Dr Rafael Yáñez-Muñoz, from the School of Biological Sciences at Royal Holloway, University of London, is leading a team of researchers working to develop a novel treatment for spinal cord injury - which leaves sufferers with devastating, life-long effects including paralysis. [More]
State highlights: Calif. ballot measure updates; Va. prison health care budget shortfall

State highlights: Calif. ballot measure updates; Va. prison health care budget shortfall

A selection of health policy stories from California, Arizona, Maine, South Dakota, Virginia, Maryland, Louisiana, Pennsylvania, Massachusetts, North Carolina, Texas, Washington state and Missouri. [More]
New genetic guideline to help physicians make right diagnosis for subtypes of muscular dystrophy

New genetic guideline to help physicians make right diagnosis for subtypes of muscular dystrophy

The American Academy of Neurology (AAN) and the American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) offer a new guideline on how to determine what genetic tests may best diagnose a person's subtype of limb-girdle or distal muscular dystrophy. [More]
LCSB scientists characterize new tumour suppressor gene

LCSB scientists characterize new tumour suppressor gene

Scientists at the Luxembourg Centre for Systems Biomedicine of the University of Luxembourg have published their findings that mutations in a gene known as "ARMC5" promote the growth of benign tumours in the adrenal glands and on the meninges: ARMC5 appears to belong to the group of so-called tumour suppressor genes. [More]
New guideline makes recommendations about treating, managing distal muscular dystrophy

New guideline makes recommendations about treating, managing distal muscular dystrophy

A new guideline from the American Association of Neuromuscular & Electrodiagnostic Medicine and the American Academy of Neurology recommends guidance on how doctors should evaluate the full picture—from symptoms, family history, and ethnicity, to a physical exam and certain lab test results—in order to determine what genetic tests may best diagnose a person's subtype of limb-girdle or distal muscular dystrophy. [More]
Isis Pharmaceuticals provides update on Phase 2 clinical studies of ISIS-SMN Rx in children with SMA

Isis Pharmaceuticals provides update on Phase 2 clinical studies of ISIS-SMN Rx in children with SMA

Isis Pharmaceuticals, Inc. today provided an update on its ongoing open-label Phase 2 clinical studies of ISIS-SMN Rx in infants and children with spinal muscular atrophy at the 19th International World Muscle Society (WMS) Congress in Berlin, Germany. [More]
Researchers develop potential fast-acting "vaccine" for myasthenia gravis

Researchers develop potential fast-acting "vaccine" for myasthenia gravis

Nearly 60,000 Americans suffer from myasthenia gravis (MG), a non-inherited autoimmune form of muscle weakness. The disease has no cure, and the primary treatments are nonspecific immunosuppressants and inhibitors of the enzyme cholinesterase. [More]

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]