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.

FKF receives funds to launch Fragile Kids Partner Program

The Centene Foundation for Quality Healthcare announced today that it has awarded a $35,000 grant to Fragile Kids Foundation, Inc. (FKF), a nonprofit organization based in Atlanta, Ga.

12 Feb 2010

Women riders support MDA's initiative on neuromuscular disease

During this year's 69th Annual Daytona Bike Week, hundreds of female motorcycle enthusiasts and Harley-Davidson Motor Company are banding together to support the Muscular Dystrophy Association (MDA) and their efforts to find a cure for neuromuscular disease.

6 Feb 2010

Tβ4 may help treat skeletal muscle disease in Duchenne muscular dystrophy

REGENERX BIOPHARMACEUTICALS, INC. announced today that a research team in Washington, D.C. has found that dystrophin-deficient Mdx mice, treated twice a week for six months with Tβ4, showed a significant increase in skeletal muscle regenerating fibers compared to untreated mice. No effects related to muscle function or fibrosis and no adverse reactions were observed in the mice.

6 Feb 2010

BioMarin announces update on BMN 110 Phase I/II trial for Morquio A Syndrome

BioMarin Pharmaceutical Inc. today announced an update on the Phase I/II trial for BMN 110 or N-acetylgalactosamine 6-sulfatase (GALNS), intended for the treatment of the lysosomal storage disorder Mucopolysaccharidosis Type IVA (MPS IVA), or Morquio A Syndrome. Preliminary clinical data from the first 24 weeks of the study (12 weeks at 0.1mg/kg and 12 weeks at 1.0 mg/kg) have been evaluated, and BioMarin plans to announce top-line results for the full 36-week study after completion of dosing at 2.0 mg/kg in the second quarter of 2010.

5 Feb 2010

BioMarin Pharmaceutical signs stock purchase agreement to acquire LEAD Therapeutics

BioMarin Pharmaceutical Inc. announced today that it has entered into a stock purchase agreement to acquire LEAD Therapeutics, Inc. (LEAD), a small private drug discovery and early stage development company with key compound LT-673, an orally available poly (ADP-ribose) polymerase (PARP) inhibitor for the treatment of patients with rare, genetically defined cancers.

4 Feb 2010

Ambassadors of Purple selected to help promote Purple Day for Epilepsy

As the third annual, worldwide Purple Day for Epilepsy approaches on March 26, 2010, organizers in Canada and in 15 other participating countries around the globe are looking for volunteers to promote epilepsy awareness in local communities.

3 Feb 2010

ALS TDI and Oxford BioMedica extend collaboration to develop gene therapies for Lou Gehrig's disease

The ALS Therapy Development Institute and Oxford BioMedica announced today the extension of their collaboration following successful completion of the first phase. The extended collaboration, which is funded by the Muscular Dystrophy Association, aims to advance the development of Oxford BioMedica's preclinical gene therapy candidate, MoNuDin®, and to evaluate other gene-based strategies for the treatment of amyotrophic lateral sclerosis (ALS or Lou Gehrig's disease).

28 Jan 2010

UF researchers work out to find new treatments for Pompe disease

As scientists work to find new treatments for Pompe disease - the devastating genetic "villain" that drives the efforts of the main characters in the new film "Extraordinary Measures" - University of Florida researchers are hopeful that gene therapy will help patients in the late stages of the disease breathe on their own.

27 Jan 2010

UCSB research: Rapamycin drug, a potential treatment for ADPKD

Research performed at UC Santa Barbara points to the drug rapamycin as a potential treatment for kidney disease. The study builds on past research and shows that studies performed on mice are more likely to translate to humans than previously thought.

27 Jan 2010

Loss of key proteins may account for molecular abnormalities associated with myotonic dystrophy

Research on the genetic defect that causes myotonic muscular dystrophy has revealed that the mutation disrupts an array of metabolic pathways in muscle cells through its effects on two key proteins.

24 Jan 2010

Study identifying new form of adult onset muscular dystrophy published

A study published in today's online edition the American Journal of Human Genetics, allowed the first identification of a new form of adult onset muscular dystrophy.

22 Jan 2010

PTC Therapeutics initiates additional clinical trial of ataluren in boys and young men with nmDBMD

PTC Therapeutics, Inc. (PTC) today announced the initiation of an additional clinical trial of ataluren (PTC124®) in boys and young men with nonsense mutation Duchenne/Becker muscular dystrophy (nmDBMD) who have permanently lost the ability to walk independently.

20 Jan 2010

A novel approach for funding children's genetic disease research

Parent Project Muscular Dystrophy together with Trisomy 18 Foundation are among 100 charities that earned a $25,000 grant during Round 1 of the Chase Community Giving Challenge on Facebook.

19 Jan 2010

New type of fat-producing cells may speed up treatments for muscular dystrophy and fibrosis

A team of University of British Columbia researchers has identified fat-producing cells that possess "dual-personalities" and may further the development of treatments for muscle diseases such as muscular dystrophy and fibrosis.

19 Jan 2010

Link between dystrophin gene and age of cardiac disease onset in BMD patients identified

Investigators in The Research Institute at Nationwide Children's Hospital have identified a link between specific modifications of the dystrophin gene and the age of cardiac disease onset in patients with Becker muscular dystrophy (BMD). This information could help clinicians provide early cardiac intervention for BMD patients based on genetic testing results performed on a blood sample. These findings are a result of analysis of the largest number of BMD patients to date and are published in the December issue of the journal Circulation: Cardiovascular Genetics.

18 Jan 2010

Flexpoint to develop infant bed cover using patented Bend Sensor® design

Flexpoint Sensor Systems, Inc. announced that they are working on the development of an infant bed cover using their patented Bend Sensor® design. The mattress will be used to monitor infants in the prevention of sudden infant death syndrome (“SIDS”).

18 Jan 2010

New Jersey to legalize medical marijuana

The New York Times: "The New Jersey Legislature approved a measure on Monday that would make the state the 14th in the nation, but one of the few on the East Coast, to legalize the use of marijuana to help patients with chronic illnesses.

13 Jan 2010

BioMarin Pharmaceutical commences BMN 195 Phase 1 clinical study for DMD

BioMarin Pharmaceutical Inc. announced today that the first subject has initiated treatment in the Phase 1 clinical study of BMN 195, a small molecule utrophin upregulator, for the treatment of Duchenne muscular dystrohpy (DMD). Initial top-line results are expected in the third quarter of 2010.

12 Jan 2010

Jiangsu Government grants $1.8M for research and development of hUC-MSC technologies

The Jiangsu Government's Science and Technology Department has announced a $1.8 million grant - the Jiangsu Technological Achievements Transformational Grant - to support the research and development of human umbilical cord mesenchymal stem cell (hUC-MSC) technologies. The grant recipient and administrator, Shenzhen Beike Biotechnology Co., Ltd. (http://www.beikebiotech.com ), is working with Jiangsu University and Nanjing University's Drum Tower Hospital to fulfill the grant's requirements.

9 Jan 2010

Scientists identify gene underlying a disease that causes temporary paralysis of skeletal muscle

Scientists have identified a gene underlying a disease that causes temporary paralysis of skeletal muscle. The finding, they say, illustrates how investigations of rare genetic diseases can drive insights into more common ones.

8 Jan 2010