Duchenne Muscular Dystrophy News and Research

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Duchenne muscular dystrophy (DMD) is a progressive muscle disorder that causes the loss of both muscle function and independence. DMD is perhaps the most prevalent of the muscular dystrophies and is the most common lethal genetic disorder diagnosed during childhood today. Each year, approximately 20,000 children worldwide are born with DMD (one of every 3,500 male children).

Immune reaction to dystrophin may contribute to muscle disease: Study

An immune reaction to dystrophin, the muscle protein that is defective in patients with Duchenne muscular dystrophy, may pose a new challenge to strengthening muscles of patients with this disease, suggests a new study appearing in the October 7, 2010, issue of The New England Journal of Medicine.

7 Oct 2010

Patients with muscular dystrophy mount immune response to dystophin protein prior to gene therapy: Study

A clinical trial designed to replace the genetic defect causing the most common form of muscular dystrophy has uncovered an unexpected aspect of the disease. The trial, based on therapy designed by scientists at the University of North Carolina at Chapel Hill School of Medicine, showed that some patients mount an immune response to the dystrophin protein even before they have received the gene therapy.

7 Oct 2010

NIH awards three grants to explore novel treatment strategies for muscular dystrophy

Three grants totaling more than $4.5 million, from agencies of the National Institutes of Health, will be used to explore novel treatment strategies for muscular dystrophy.

30 Sep 2010

FDA designates AMT-080 gene therapy for Duchenne muscular dystrophy

Amsterdam Molecular Therapeutics (AMT) Holding N.V. a leader in the field of human gene therapy, announced today that the U.S. Food and Drug Administration (FDA) has designated AMT-080, a gene therapy for Duchenne muscular dystrophy (DMD) as an orphan drug.

24 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

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

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

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

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

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

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

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

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

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

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