Muscular Dystrophy News and Research

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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.

Venom protein could treat Duchenne muscular dystrophy

While Spider-Man is capturing the imagination of theatergoers, real-life spider men in Upstate New York are working intently to save a young boy's life. It all began in 2009, when Jeff Harvey, a stockbroker from the Buffalo suburbs, discovered that his grandson, JB, had Duchenne muscular dystrophy. The disease is fatal. It strikes only boys, causing their muscles to waste away.

17 Jul 2012

UMMS scientists identify new genetic mutation that causes familial ALS

A new genetic mutation that causes familial amyotrophic lateral sclerosis (ALS), a fatal neurological disorder also known as Lou Gehrig's Disease, has been identified by a team of scientists led by researchers at the University of Massachusetts Medical School (UMMS).

16 Jul 2012

New eye-tracking device for amputees

Millions of people suffering from Multiple Sclerosis, Parkinson's, muscular dystrophy, spinal cord injuries or amputees could soon interact with their computers and surroundings using just their eyes, thanks to a new device that costs less than £40.

16 Jul 2012

Researchers link newly discovered gene mutations to ALS

Researchers have linked newly discovered gene mutations to some cases of the progressive fatal neurological disease amyotrophic lateral sclerosis - ALS, also known as Lou Gehrig's disease. Shedding light on how ALS destroys the cells and leads to paralysis, the researchers found that mutations in this gene affect the structure and growth of nerve cells.

16 Jul 2012

Communication between brain and muscle cells is short-lived without LRP4 protein

Communication between the brain and muscle must be strong for us to eat, breathe or walk. Now scientists have found that a protein known to be on the surface of muscle cells must be present in both tissues to ensure the conversation is robust.

12 Jul 2012

Read-through molecule improves muscle function in DMD

A compound that results in a read-through on nonsense mutations in messenger RNA restores dystrophin expression in an animal model of Duchenne muscular dystrophy, research shows.

9 Jul 2012

Novel gene therapy approach may treat dysferlinopathies

The challenge of treating patients with genetic disorders in which a single mutated gene is simply too large to be replaced using traditional gene therapy techniques may soon be a thing of the past. A Nationwide Children's Hospital study describes a new gene therapy approach capable of delivering full-length versions of large genes and improving skeletal muscle function. The strategy may hold new hope for treating dysferlinopathies and other muscular dystrophies.

9 Jul 2012

Scientists transplant stem cells from patients with limb-girdle muscular dystrophy into mice

Stem cells from patients with a rare form of muscular dystrophy have been successfully transplanted into mice affected by the same form of dystrophy, according to a new study published today in Science Translational Medicine.

28 Jun 2012

New compound could treat certain types of genetic disorders in muscles

Scientists at UCLA have identified a new compound that could treat certain types of genetic disorders in muscles. It is a big first step in what they hope will lead to human clinical trials for Duchenne muscular dystrophy.

28 Jun 2012

First clinical trial to test cardiac drug regimen for DMD

The first landmark randomized clinical trial for a cardiac drug regimen in Duchenne muscular dystrophy (DMD) is testing whether earlier treatment can stop or slow down heart damage that usually kills people with the disease.

27 Jun 2012

PPMD to host first Transforming Duchenne Care Meeting from June 27-28

Parent Project Muscular Dystrophy (PPMD) will host the first Transforming Duchenne Care Meeting, June 27-28, 2012, immediately preceding the Annual Connect Conference in Fort Lauderdale, Florida.

26 Jun 2012

PPMD, AVI to participate in Rock 'n' Roll Seattle Marathon & Half Marathon

AVI BioPharma is teaming up with Parent Project Muscular Dystrophy (PPMD)'s endurance program, Run For Our Sons, to participate in the Rock 'n' Roll Seattle Marathon & Half Marathon on June 23, 2012. The goal is to raise money and awareness to help end Duchenne muscular dystrophy (Duchenne), the most common form of muscular dystrophy.

21 Jun 2012

Decision Resources forecasts the launch of four new agents to treat DMD by 2021

Decision Resources, one of the world's leading research and advisory firms focusing on pharmaceutical and healthcare issues, finds that interviewed experts unanimously identify a disease-modifying therapy as the highest unmet need in the treatment of Duchenne muscular dystrophy (DMD).

8 Jun 2012

CDI introduces MyCell Services

Cellular Dynamics International, Inc. (CDI), the world's largest commercial producer of human induced pluripotent stem (iPS) cell lines and tissue cells, today announced the launch of its MyCell Services.

7 Jun 2012

Vejthani Hospital offers most effective procedure in gender selection

Vejthani Hospital Bangkok recommends a gender selection procedure for a couple who desire to choose a child's gender at Vejthani ART Center.

16 May 2012

Study identifies how skeletal muscle stem cells respond to muscle injury

A study conducted by Children's Hospital & Research Center Oakland scientists identifies how skeletal muscle stem cells respond to muscle injury and may be stimulated to improve muscle repair in Duchenne Muscular Dystrophy, a severe inherited disease of muscle that causes weakness, disability and, ultimately, heart and respiratory failure.

15 May 2012

DYNC1H1 genetic mutation contributes to SMA-LED

Scientists have confirmed that mutations of a gene are responsible for some cases of a rare, inherited disease that causes progressive muscle degeneration and weakness: spinal muscular atrophy with lower extremity predominance, also known as SMA-LED.

11 May 2012

New measurement tool for clinical trials to help children with Charcot-Marie-Tooth disease

An international study led by the University of Sydney and published in the Annals of Neurology has the potential to improve the design of clinical trials for the treatment of Charcot-Marie-Tooth disease, a disorder which affects the peripheral nervous system.

8 May 2012

Research grants on facioscapulohumeral dystrophy prove useful as genetic basis is found

Businessman Bill Moss’s determination to fight his untreatable muscle-wasting disease may have paid off. Four years ago, Mr Moss, a former Macquarie Bank Executive Director, established a charitable research foundation, FSHD Global, which partly funded the Italian researchers who made the breakthrough.

8 May 2012

Human stem cells effective in treating muscular dystrophy

Researchers from the University of Minnesota's Lillehei Heart Institute have effectively treated muscular dystrophy in mice using human stem cells derived from a new process that - for the first time - makes the production of human muscle cells from stem cells efficient and effective.

5 May 2012