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.

OSC and RINCH offer support to speed discovery techniques of childhood diseases

Worried about her high fever and severe abdominal pain, a young couple rushed their baby daughter to the emergency department of Nationwide Children's Hospital. Physicians there found a lump in her belly, and, after examining X-rays and blood work, confirmed the parents' worst fear: their 18-month-old little girl had neuroblastoma, a rare pediatric cancer that involves the adrenal glands.

20 Jan 2011

GSK and Prosensa commence GSK2402968 Phase III study in DMD

GlaxoSmithKline and Prosensa today announced that the first patient has commenced treatment in the Phase III clinical study investigating GSK2402968, in ambulant boys with Duchenne Muscular Dystrophy (DMD), who have a dystrophin gene mutation amenable to an exon 51 skip (up to 13% of boys with DMD). Commencement of this study confirms previously announced plans to progress this asset into Phase III.

20 Jan 2011

University of Minnesota places order for Bruker's ultra-high field NMR spectrometers

Bruker announces an order valued at over $7.5 million from the University of Minnesota for its new Nuclear Magnetic Resonance (NMR) laboratory, including ultra-high field Avance™ III 900 and Avance™ III 850 NMR spectrometers. The systems will be installed in the newly constructed and greatly expanded NMR Facility at the University of Minnesota.

12 Jan 2011

Genethon deploys Atempo Digital Archive to optimize biotherapy research data storage resources

Atempo, a leading provider of cross-platform data protection and archiving solutions, announced today that Genethon, a French non-profit biotherapy research center, has deployed Atempo Digital Archive, Atempo's advanced file archiving platform, to optimize its storage resources.

12 Jan 2011

AMT enters agreement with Consortium for clinical development of novel gene therapy to treat Sanfilippo B

Amsterdam Molecular Therapeutics, a leader in human gene therapy, announced today that it has entered into an agreement with Institut Pasteur, Paris, France, and a group of French research institutes (together the "Consortium") to support clinical development of a novel gene therapy to treat Sanfilippo B.

10 Jan 2011

Acceleron recieves $1.5 million MDA grant for ACE-031 clinical study in Duchenne Muscular Dystrophy

Acceleron Pharma, 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 has been awarded a $1.5 million grant from the Muscular Dystrophy Association to support clinical studies of ACE-031 in Duchenne Muscular Dystrophy, a disabling neuromuscular disease in which patients experience a progressive loss of muscle mass and strength.

6 Jan 2011

Encouraging results from exon skipping gene-based therapy study for DMD

Scientists at Royal Holloway, University of London have reported encouraging results in a new gene-based therapy for Duchenne Muscular dystrophy (DMD) which at present has no known cure and affects one in 3,000 young boys.

4 Jan 2011

Biglycan protein offers potential therapy for muscular dystrophy

A novel potential therapy based on a natural human protein significantly slows muscle damage and improves function in mice who have the same genetic mutation as boys with the most common form of muscular dystrophy, according to a paper published online Dec. 27 in the Proceedings of the National Academy of Sciences.

28 Dec 2010

Scientists boost protein supply to enhance muscle function, reduce nerve-dependent fatigue in mice

Researchers at Vanderbilt University have "engineered" a mouse that can run on a treadmill twice as long as a normal mouse by increasing its supply of acetylcholine, the neurotransmitter essential for muscle contraction.

21 Dec 2010

MDA awards Repligen $1.4M for research of new compound with therapeutic potential for SMA

The Muscular Dystrophy Association today announced it has awarded $1.4 million in new research funding to the biopharmaceutical firm Repligen of Waltham, Mass., to complete the preclinical work needed to begin human clinical trials of a promising therapeutic compound for spinal muscular atrophy (SMA).

17 Dec 2010

MDA supports Repligen with $1.4M grant for RG3039 Spinal Muscular Atrophy development program

Repligen Corporation today announced that the Company has received $1,400,000 in research funding from the Muscular Dystrophy Association ("MDA") to support the ongoing development of RG3039 for Spinal Muscular Atrophy ("SMA"). RG3039, our lead compound, is an inhibitor of an RNA processing enzyme which targets increased production of SMN, a protein of deficient levels in patients with SMA.

15 Dec 2010

VCP gene causes some instances of familial ALS: Researchers

Using a new gene sequencing method, a team of researchers led by scientists from Johns Hopkins and the National Institutes of Health has discovered a gene that appears to cause some instances of familial amyotrophic lateral sclerosis. The finding could lead to novel ways to treat the more common form of this fatal neurodegenerative disease, which kills the vast majority of the nearly 6,000 Americans diagnosed with ALS every year.

10 Dec 2010

First standard-of-care guidelines for children with congenital muscular dystrophy

In 2008, Eunice and Andrew Kim were facing a situation that would bewilder any parents: Two expert pediatric surgeons were giving opposing advice about treatment for their daughter.

8 Dec 2010

Beike exits stem cell joint venture with SiriCell

Shenzhen Beike Biotechnology Co. Ltd. ("Beike") and SiriCell Technologies, Inc. announced that they are ending their partnership in "Beike Holdings Limited", a joint venture formed by the two companies in 2008. Going forward, Beike will exit the joint venture, and all current and future Beike Holdings-initiated business activities will operate exclusively under the SiriCell brand. Beike Holdings was established to expand the availability of Beike's stem cell products outside of China through collaborative hospital partnerships and new international subsidiaries.

8 Dec 2010

MU researcher hopes to protect cardiac muscles for children with DMD

Children with Duchenne muscular dystrophy face a future of rapidly weakening muscles, which usually leads to death by respiratory or cardiac failure before their 30th birthday. While researchers are hopeful that gene therapy could eventually evolve into an effective treatment, few have targeted the heart of the problem as much as Dongsheng Duan, PhD.

7 Dec 2010

Pharnext receives French approval for Pleodrug Phase II clinical trial in Charcot-Marie Tooth disease type 1A

Pharnext SAS, a biopharmaceutical company specializing in the development of innovative treatments for severe neurological diseases and other major unmet medical needs, today announced that the French Agency for Healthcare Product Safety (AFSSAPS) has authorized a Phase II clinical trial with the first drug (a Pleodrug™) generated by the company's Pleotherapy™ technology platform in patients suffering from Charcot-Marie Tooth disease type 1A (CMT1A.

4 Dec 2010

AMT, Amgen amend GDNF gene license agreement

Amsterdam Molecular Therapeutics Holding N.V., a leader in the development of gene-based therapies, today announced that it has amended and restated its licensing agreement with Amgen for gene therapy applications incorporating the GDNF (glial cell derived neurotrophic factor) gene, to which Amgen holds rights. Financial terms were not disclosed.

2 Dec 2010

Researchers investigate ways to counteract unhealthy effects of fat

A fat yet muscular mouse is helping researchers learn whether more muscle improves the cardiovascular health of obese individuals. We are looking for ways to counteract the unhealthy effects of fat, said Dr. David Stepp, vascular biologist at the Medical College of Georgia Vascular Biology Center and co-director of MCG's Diabetes & Obesity Discovery Institute.

23 Nov 2010

Lotsa Helping Hands' new feature connects community members to healthcare nonprofit organizations

Lotsa Helping Hands, the leading provider of a web-based caregiving coordination service, today announces a new feature that connects its community members to trusted resources from many of the nation's leading healthcare nonprofit organizations.

20 Nov 2010

New editorial dispel doubts about Lou Gehrig's ALS diagnosis

A recent study suggesting that amyotrophic lateral sclerosis (ALS) may be attributed to repetitive head trauma experienced in collision sports lacks scientific epidemiological evidence to support this claim.