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.

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

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.

18 Aug 2010

AMT's MAA for LPLD gene therapy product Glybera on schedule

Amsterdam Molecular Therapeutics, a leader in the field of human gene therapy, today announced that the Marketing Authorisation Application (MAA) for Glybera(R) remains on schedule following meetings with the European Medicine Agency (EMA) concerning the Day 120 List of Questions. The company is confident that Glybera(R), a gene therapy product for lipoprotein lipase deficiency (LPLD), remains on track for a regulatory decision by mid-2011.

18 Aug 2010

Columbus hospital receives PPMD grant to conduct clinical testing for Duchenne

Parent Project Muscular Dystrophy, the largest non-profit organization in the United States focused on finding a cure for Duchenne muscular dystrophy (Duchenne), will award a $600,000 grant to Nationwide Children's Hospital in Columbus, Ohio to conduct clinical testing of a promising gene therapy technique for muscle disease.

10 Aug 2010

Myths and realities of Medicare bidding program for durable medical equipment

Providers of home medical equipment and services across California are proposing a fiscally responsible alternative to the mislabeled "competitive" bidding scheme currently under way in Medicare that will actually discourage competition, reduce access to care for many of California's 4.6 million Medicare beneficiaries, and put thousands of California homecare providers out of business.

7 Aug 2010

Acceleron's ACE-031 protein therapeutic for DMD receives FDA Fast Track designation

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 it received FDA Fast Track designation for ACE-031 for the treatment of Duchenne Muscular Dystrophy (DMD), a fatal neuromuscular disease in which patients suffer from the progressive loss of muscle mass and strength. ACE-031 is an investigational protein therapeutic being developed to increase muscle mass and strength.

5 Aug 2010

Insmed second-quarter net loss decreases to $0.4 million

Insmed Inc., a biopharmaceutical company, today reported results for the second quarter and six-months ended June 30, 2010.

4 Aug 2010

U.S. clinical trials provide further evidence of importance of storing umbilical cord blood

Cord Blood America, Inc., the umbilical cord blood stem cell preservation company focused on bringing the life saving potential of stem cells, a biological insurance policy, to families nationwide and internationally, today said that clinical trials ongoing in the U.S. using umbilical cord blood in the treatment of cerebral palsy, high-risk hematologic cancer, injured spinal cords and Type 1 diabetes provide significant further evidence of the importance of storing umbilical cord blood at the time of birth.

3 Aug 2010

BioMarin reports $0.5 million GAAP net loss for second-quarter 2010

BioMarin Pharmaceutical Inc. today announced financial results for the second quarter of 2010. GAAP net loss was $0.5 million ($0.01 per diluted share) for the second quarter of 2010, compared to GAAP net income of $1.3 million ($0.01 per diluted share) for the second quarter of 2009. Non-GAAP net income was $8.6 million ($0.08 per diluted share) for the second quarter of 2010, compared to non-GAAP net income of $9.0 million ($0.09 per diluted share) for the second quarter of 2009.

3 Aug 2010

BioMarin completes BMN 195 Phase 1 clinical trial for Duchenne muscular dystrophy

BioMarin Pharmaceutical Inc. announced today that it has completed the Phase 1 clinical study of BMN 195, a small molecule utrophin up-regulator, for the treatment of Duchenne muscular dystrophy (DMD). The Phase 1 clinical trial was a single-center, double-blind, placebo-controlled, single-dose escalation study followed by a multiple-dose escalation study in healthy volunteers.

3 Aug 2010

Patient advocacy groups, home medical associations call for end to Medicare competitive bidding program

In a letter signed by 58 patient advocacy groups and home medical equipment associations, the American Association for Homecare urged Congress to pass H.R. 3790, a bill to repeal the Medicare competitive bidding program for durable medical equipment in a budget-neutral manner. The bill currently has 252 cosponsors in the U.S. House of Representatives.

17 Jul 2010

PPMD to provide grant to clinical sites participating in Phase 2 study of ACE-031

Patricia A. Furlong, Founding President and CEO of Parent Project Muscular Dystrophy, the largest non-profit organization in the United States focused on finding a cure for Duchenne muscular dystrophy, announced that PPMD will provide a grant to clinical sites participating in Acceleron Pharma's Phase 2 clinical study of ACE-031. These grants will help patients' families cover travel costs to and from the study site, and hotel rooms and meals during study visits.

15 Jul 2010

Children's National and George Washington University receive NIH Clinical and Translational Science Award

Children's National Medical Center, in partnership with The George Washington University Medical Center, has received a prestigious Clinical and Translational Science Award (CTSA) from the National Center for Research Resources of the National Institutes of Health.

15 Jul 2010

CSHL scientists reverse symptoms of Type III SMA using ASOs

The devastating, currently incurable motor-neuron disease spinal muscular atrophy might soon be treated with tiny, chemically modified pieces of RNA called antisense oligonucleotides.

13 Jul 2010

New treatments to alleviate symptoms of muscular dystrophy, depression and dementia

Scientists researching a toxin extracted from the venom of the honey bee have used this to inform the design of new treatments to alleviate the symptoms of conditions such as muscular dystrophy, depression and dementia.

12 Jul 2010

Scientists identify potential disease genes in humans and mice

An international consortium of scientists of Helmholtz Zentrum München and the University of Toronto has identified previously unknown potential disease genes in humans and mice. Genes on the X chromosome, which regulate embryonic development, are the focus of the current publication in the renowned journal Genome Research. Men have only one X chromosome, and therefore mutations on this chromosome disproportionately affect males, frequently leading to serious diseases such as hemophilia, muscular dystrophy and mental retardation.

7 Jul 2010

Simple blood test may help detect chromosomal abnormalities in developing foetus

Researchers in The Netherlands believe they are on the verge of developing a simple, prenatal blood test that would be able to detect accurately chromosomal abnormalities in the developing foetus.

29 Jun 2010

Non-Invasive prenatal blood test to detect chromosomal abnormalities in foetus

Researchers in The Netherlands believe they are on the verge of developing a simple, prenatal blood test that would be able to detect accurately chromosomal abnormalities in the developing foetus. At present, the only reliable way to do this is through amniocentesis or chorionic villus sampling, both of which are invasive and carry the risk of triggering a miscarriage.

29 Jun 2010

Research findings suggest possible diagnostic test for ALS

A team led by a Los Angeles Biomedical Research Institute researcher developed a novel approach to efficiently identify disease relevant genes in human complex neurological disorders and demonstrated the effectiveness of this method by applying it to Amyotrophic Lateral Sclerosis, or Lou Gehrig's Disease, according to a new study reported online in the journal, Human Molecular Genetics.

25 Jun 2010

Prosensa, GSK initiate additional programs for DMD

Prosensa, the Dutch biopharmaceutical company focusing on RNA modulating therapeutics, announces the initiation of two further programmes under its existing alliance with GSK covering novel RNA-based treatments for Duchenne Muscular Dystrophy (DMD). The initiation of these additional programs under the terms of the existing alliance agreement is a validation of both the potential of Prosensa's "exon skipping" platform and the ongoing relationship.

23 Jun 2010

Researchers identify promising gene therapy for DMD

CureDuchenne, the Orange County non-profit focused on finding a cure for Duchenne muscular dystrophy (DMD) is one step closer to that goal through the work of Dr. Jeff Chamberlain.

23 Jun 2010

Muscular Dystrophy News and Research

Further Reading

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

AMT's MAA for LPLD gene therapy product Glybera on schedule

Columbus hospital receives PPMD grant to conduct clinical testing for Duchenne

Myths and realities of Medicare bidding program for durable medical equipment

Acceleron's ACE-031 protein therapeutic for DMD receives FDA Fast Track designation

Insmed second-quarter net loss decreases to $0.4 million

U.S. clinical trials provide further evidence of importance of storing umbilical cord blood

BioMarin reports $0.5 million GAAP net loss for second-quarter 2010

BioMarin completes BMN 195 Phase 1 clinical trial for Duchenne muscular dystrophy

Patient advocacy groups, home medical associations call for end to Medicare competitive bidding program

PPMD to provide grant to clinical sites participating in Phase 2 study of ACE-031

Children's National and George Washington University receive NIH Clinical and Translational Science Award

CSHL scientists reverse symptoms of Type III SMA using ASOs

New treatments to alleviate symptoms of muscular dystrophy, depression and dementia

Scientists identify potential disease genes in humans and mice

Simple blood test may help detect chromosomal abnormalities in developing foetus

Non-Invasive prenatal blood test to detect chromosomal abnormalities in foetus

Research findings suggest possible diagnostic test for ALS

Prosensa, GSK initiate additional programs for DMD

Researchers identify promising gene therapy for DMD

Sensing a Healthier Future

Achieving and maintaining ISO/IEC 27001 and BS 10012 certifications

Expanding research and clinical options for children with cancer

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