Muscular Dystrophy News and Research

RSS

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 points to unique therapeutic approach for managing muscle-wasting conditions

New findings on why skeletal muscle stem cells stop dividing and renewing muscle mass during aging points up a unique therapeutic opportunity for managing muscle-wasting conditions in humans, says a new University of Colorado Boulder study.

17 Feb 2014

Prothelia, University of Nevada, Reno and Alexion partner to develop Laminin-111 for MDC1A

Prothelia Incorporated and University of Nevada, Reno announce that they have entered into strategic agreements with Alexion for the development of Laminin-111, a patented experimental protein replacement therapy for merosin-deficient congenital muscular dystrophy (MDC1A), a life-threatening, ultra-rare disease caused by a genetic deficiency of the Laminin-211 protein.

13 Feb 2014

LA Fitness to host in-club Group Fitness class in support of Augie's Quest

In support of Augie's Quest, LA Fitness announced today that it will host an in-club Group Fitness class at more than 500 of the company's locations on February 22, 2014. Augie's Quest is a nonprofit research initiative dedicated to finding treatments and a cure for amyotrophic lateral sclerosis (ALS). LA Fitness has donated to Augie's Quest for several years, and this year's fundraiser marks the fourth in-club event the company has held in support of this worthy cause.

13 Feb 2014

Researchers demonstrate new approach to treating muscular dystrophy

Researchers at Washington University School of Medicine in St. Louis have demonstrated a new approach to treating muscular dystrophy. Mice with a form of this muscle-weakening disease showed improved strength and heart function when treated with nanoparticles loaded with rapamycin, an immunosuppressive drug recently found to improve recycling of cellular waste.

12 Feb 2014

New approach for reducing levels of toxic protein fragments associated with Huntington's disease

An innovative therapeutic strategy for reducing the levels of toxic protein fragments associated with Huntington's disease uses a new approach called exon skipping to remove the disease-causing component of the essential protein, huntingtin.

11 Feb 2014

Researchers examine how toxin triggers motor neuron death

In most cases of amyotrophic lateral sclerosis (ALS), or Lou Gehrig's disease, a toxin released by cells that normally nurture neurons in the brain and spinal cord can trigger loss of the nerve cells affected in the disease, Columbia researchers reported today in the online edition of the journal Neuron.

7 Feb 2014

Study offers new avenue to pursue in quest for desperately needed treatments for ALS

St. Jude Children's Research Hospital scientists led a study showing that mutations in a gene responsible for amyotrophic lateral sclerosis (ALS) disrupt the RNA transport system in nerve cells.

6 Feb 2014

Researchers develop valid humanized model for facioscapulohumeral muscular dystrophy

Researchers at the Kennedy Krieger Institute recently announced study findings showing the successful development of a humanized preclinical model for facioscapulohumeral muscular dystrophy (FSHD), providing scientists with a much needed tool to accelerate novel therapeutic research and development.

28 Jan 2014

Jackson Laboratory associate professor receives grant to study gene regulation at RNA level

Jackson Laboratory Associate Professor Jeffrey Chuang, Ph.D., has been awarded a two-year grant totaling $519,750 from the National Human Genome Research Institute for his studies of how RNA (molecules vital to protein formation in cells) interacts with proteins to change how genes are expressed.

27 Jan 2014

Researcher finds possible way to unravel misfolded proteins by reprogramming yeast protein

At the heart of brain diseases such as amyotrophic lateral sclerosis, Alzheimer's disease, and Parkinson's disease is protein misfolding, in which distorted proteins are unable to perform their normal functions. At present, there is no known way to reverse protein misfolding.

17 Jan 2014

Scientists reveal atomic-level view of genetic defect that causes myotonic dystrophy type 2

Scientists from the Florida campus of The Scripps Research Institute have revealed an atomic-level view of a genetic defect that causes a form of muscular dystrophy, myotonic dystrophy type 2, and have used this information to design drug candidates with potential to counter those defects—and reverse the disease.

6 Jan 2014

Electromed gets FDA clearance to market next generation SmartVest Airway Clearance System

Electromed, Inc., a global medical device company, today announced it has received notification from the U.S. Food and Drug Administration that its next generation SmartVest Airway Clearance System, the model SQL has been cleared to market.

30 Dec 2013

Scientists develop new potential cure for rare eye disease

University of British Columbia and Vancouver Coastal Health scientists have developed a potential cure for a rare eye disease, showing for the first time that a drug can repair a birth defect.

22 Dec 2013

New MDA Endurance Program aims to find treatments and cures for muscle disease

The Muscular Dystrophy Association today announced the formation and launch of MDA Team Momentum, a new nationwide endurance program designed to help support MDA's mission to find treatments and cures for muscle disease.

20 Dec 2013

New gene mutation helps doctors give more accurate diagnosis of mitochondrial myopathy

A new gene mutation which will help doctors give a more accurate diagnosis of a particular type of brain and muscle disease in children has been discovered for the first time by University of Leeds experts.

17 Dec 2013

Retrophin to acquire privately-held company, Kyalin Biosciences

Retrophin, Inc. today announced that it will acquire privately-held Kyalin Biosciences, Inc., an early-stage company based in San Diego, CA, that is developing therapies targeting the core symptoms of autism and related conditions.

12 Dec 2013

Retrophin signs agreement with Novartis for exclusive U.S. license for Syntocinon Nasal Spray

Retrophin, Inc. today announced that it has signed an agreement with Novartis Pharma AG for an exclusive U.S. license for Syntocinon Nasal Spray, the intranasal formulation of a synthetic version of the naturally occurring peptide hormone oxytocin, for an upfront payment of $5.0 million plus milestone payments and royalties.