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.

New study signals hope for treatment of rare neurodegenerative disease

A new study led by St. Jude Children's Research Hospital scientists signals hope for treatment of a neurodegenerative disease and a new model for understanding the mechanism at work in other more common neurodegenerative disorders.

23 Sep 2010

Specific orofacial characteristics can lead to accurate diagnosis of fetal alcohol spectrum disorders

Fetal alcohol spectrum disorders (FASD) is an umbrella term that describes the range of effects that can occur in a person whose mother drank alcohol while pregnant. Each year, FASD affect an estimated 40,000 infants in the United States—more than spina bifida, Down syndrome, and muscular dystrophy combined.

22 Sep 2010

Brown grants exclusive intellectual property license to Tivorsan Pharmaceuticals

Development of a potential treatment for the most common form of muscular dystrophy is moving into a new phase at Brown University. Brown has granted an exclusive license for the intellectual property to Tivorsan Pharmaceuticals, a startup firm that will strive to see it through human trials.

22 Sep 2010

Study shows Americans with muscular dystrophy live longer than African-Americans

A new study shows that white men and boys are living longer with muscular dystrophy due to technological advances in recent years, but that the lives of African-American men and boys with muscular dystrophy have not been extended at the same rate. The research will be published in the September 14, 2010, issue of Neurology, the medical journal of the American Academy of Neurology.

14 Sep 2010

Shire announces expansion of Human Genetic Therapies pipeline

Shire plc, the global specialty biopharmaceutical company, today announced the expansion of its Human Genetic Therapies pipeline through the exclusive license, in markets outside of North America, for the activin receptor type IIB class of molecules being developed by Acceleron Pharma Inc., a private biotechnology company based in Cambridge, Massachusetts.

10 Sep 2010

Mayo Clinic opens newly expanded neurophysiology laboratory at Florida campus

Mayo Clinic unveiled a newly expanded, state-of-the-art neurophysiology laboratory on its Florida campus to provide additional services and care for more patients, many of whom are referred by community physicians for specialized diagnostic testing.

10 Sep 2010

Acceleron Pharma and Shire announce joint development of ACE-031 for DMD

Acceleron Pharma, Inc., a biopharmaceutical company developing novel therapeutics that modulate the growth of cells and tissues including muscle, bone, fat, red blood cells and the vasculature, and Shire plc, the global specialty biopharmaceutical company, today announced a joint development and commercialization agreement for ACE-031 and other novel molecules targeting the activin receptor type IIB (ActRIIB) pathway. This pathway plays critical roles in regulating the growth of skeletal muscle.

9 Sep 2010

Research on iPS cells can imply potential cell therapy for X-linked disorders

Female induced pluripotent stem (iPS) cells, reprogrammed from human skin cells into cells that have the embryonic-like potential to become any cell in the body, retain an inactive X chromosome, stem cell researchers at UCLA have found.

4 Sep 2010

International study to improve treatments for Duchenne muscular dystrophy

A large international study aimed at improving the care of muscular dystrophy patients worldwide is being launched by physicians, physical therapists, and researchers at the University of Rochester Medical Center.

3 Sep 2010

Santhera, Ipsen partner to develop and commercialize fipamezole outside North America and Japan

Santhera Pharmaceuticals and Ipsen today announced a license agreement for the development and commercialization of fipamezole (antagonist of the adrenergic alpha-2 receptor) for territories outside of North America and Japan. This first-in-class compound is currently under investigation for the treatment of levodopa-induced dyskinesia in Parkinson's Disease.

3 Sep 2010

Study demonstrates safety of cord blood stem cell transplantation protocol to treat neurological conditions

In a new peer-reviewed article published by the Journal of Translational Medicine, scientists from Beike Biotechnology,China's leading stem cell research and regenerative medicine company, and Medistem, Inc.,reported positive safety data in 114 patients who were treated by doctors at Nanshan Affiliated Hospital of Guangdong Medical College (Shenzhen Nanshan Hospital) in Shenzhen using Beike's proprietary cord blood stem cell transplantation protocol.

3 Sep 2010

Amsterdam Molecular first half 2010 net loss in line with last year

Amsterdam Molecular Therapeutics, a leader in the field of human gene therapy, today reported its results for the first half year of 2010.

31 Aug 2010

Nationwide Children's Hospital designated as Wellstone Muscular Dystrophy Cooperative Research Center

A $7 million grant from the National Institutes of Health (NIH) will help researchers at Nationwide Children's Hospital translate new scientific findings and technological developments into novel treatments for the muscular dystrophies. The grant designates Nationwide Children's Hospital as a Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, one of three national award recipients in 2010.

27 Aug 2010

Children's Wish Foundation to grant new wishes for children diagnosed with life-threatening illness

The first word Timur ever uttered was "cat". For all of his 8 years, Timur has been fascinated by animals and dinosaurs; he loves to watch Animal Planet and the Discovery Channel. So when he was told he could have his most heartfelt wish thanks to The Children's Wish Foundation of Canada, animals were certain to be an important part of it.

23 Aug 2010

Study finds genetic change in chromosome 4 causes FSHD

The Muscular Dystrophy Association today heralds a landmark muscular dystrophy advance by an international study team of scientists and physicians from the Netherlands, United States, France and Spain. Led by MDA-grantee Silvère van der Maarel, Ph.D., at Leiden University Medical Center in the Netherlands, the collaborative study of more than 2,300 people found that two distinct genetic changes on chromosome 4 must be present to cause facioscapulohumeral muscular dystrophy (FSHD).

21 Aug 2010

FSH society applauds findings of facioscapulohumeral muscular dystrophy

This is what we have all been working towards and fighting for, says Daniel Paul Perez, co-founder, president and CEO of the FSH Society and a 48-year-old patient with facioscapulohumeral muscular dystrophy (FSHD), applauding the researchers whose study on yesterday's ScienceXpress, the journal Science's online advanced edition, pinpoints for the first time the exact genetic mechanism triggering FSHD, the most common muscular dystrophy.

21 Aug 2010

Scientists discover genetic flaw that causes facioscapulohumeral muscular dystrophy

Nearly two decades after they identified the specific genetic flaw that causes a common type of muscular dystrophy, scientists believe they have figured out how that flaw brings about the disease. The finding by an international team of researchers settles a longstanding question about the roots of facioscapulohumeral muscular dystrophy or FSHD.

20 Aug 2010

Muscular Dystrophy Association awards $14.1M in new grants

The Muscular Dystrophy Association, which has invested almost $39 million in 2010 in worldwide research seeking treatments and cures for muscle diseases, today announced that it has awarded $14.1 million in new grants.

20 Aug 2010

FDA grants Acceleron's ACE-031 orphan designation for treatment of DMD

Acceleron Pharma, Inc., a biopharmaceutical company developing novel therapeutics that modulate the growth of cells and tissues including muscle, bone, fat, red blood cells and the vasculature, today announced the United States Food and Drug Administration (FDA) granted orphan designation for ACE-031 for the treatment of Duchenne Muscular Dystrophy (DMD), a fatal neuromuscular disease in which patients experience a progressive loss of muscle mass and strength. ACE-031 is an investigational protein therapeutic being developed to increase muscle mass and strength.

20 Aug 2010

MDA awards $14.1 million in new grants to advance treatments and cures for muscle diseases

18 Aug 2010

Muscular Dystrophy News and Research

Further Reading

New study signals hope for treatment of rare neurodegenerative disease

Specific orofacial characteristics can lead to accurate diagnosis of fetal alcohol spectrum disorders

Brown grants exclusive intellectual property license to Tivorsan Pharmaceuticals

Study shows Americans with muscular dystrophy live longer than African-Americans

Shire announces expansion of Human Genetic Therapies pipeline

Mayo Clinic opens newly expanded neurophysiology laboratory at Florida campus

Acceleron Pharma and Shire announce joint development of ACE-031 for DMD

Research on iPS cells can imply potential cell therapy for X-linked disorders

International study to improve treatments for Duchenne muscular dystrophy

Santhera, Ipsen partner to develop and commercialize fipamezole outside North America and Japan

Study demonstrates safety of cord blood stem cell transplantation protocol to treat neurological conditions

Amsterdam Molecular first half 2010 net loss in line with last year

Nationwide Children's Hospital designated as Wellstone Muscular Dystrophy Cooperative Research Center

Children's Wish Foundation to grant new wishes for children diagnosed with life-threatening illness

Study finds genetic change in chromosome 4 causes FSHD

FSH society applauds findings of facioscapulohumeral muscular dystrophy

Scientists discover genetic flaw that causes facioscapulohumeral muscular dystrophy

Muscular Dystrophy Association awards $14.1M in new grants

FDA grants Acceleron's ACE-031 orphan designation for treatment of DMD

MDA awards $14.1 million in new grants to advance treatments and cures for muscle diseases

Expanding research and clinical options for children with cancer

Examining how pain could play a direct protective role in the gut

From Puberty to Menopause: Clue’s CEO, Audrey Tsang on the Power of Femtech

Latest News