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.

Beneficial effects of LifeVantage's Protandim on DMD mouse model published in Journal of Dietary Supplements

LifeVantage Corporation, the maker of Protandim®, a patented, science-based solution to oxidative stress, announced today that a peer-reviewed manuscript was published in the Journal of Dietary Supplements examining the beneficial effects of Protandim® on a mouse model of Duchenne Muscular Dystrophy (DMD), an X-linked inherited disease affecting one in 3,500 males.

22 Jun 2010

End Duchenne Day to be celebrated on June 26, 2010: PPMD

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 the Mayor of Denver, Colorado, John W. Hickenlooper, has proclaimed June 26, 2010 "End Duchenne Day" in recognition of PPMD's 16th Annual Connect Conference. Denver is playing host to the country's largest Duchenne-specific, international conference, June 24 – 27, 2010.

19 Jun 2010

CureDuchenne kicks off national fundraiser Pick Your Peak

CureDuchenne, a national 501(c)3 dedicated to raising funds and awareness in the fight against Duchenne Muscular Dystrophy, is kicking off a national fundraiser Pick Your Peak. Outdoor enthusiasts, philanthropists, families of boys with DMD, and anyone excited to get out and climb for a cause will climb the peak of their choice on June 19-20, 2010.

19 Jun 2010

UF administers Lumizyme for late-onset Pompe disease

The first commercially available treatment in the United States for patients with late-onset Pompe disease was administered Wednesday, June 16, at the University of Florida.

18 Jun 2010

UF doctors administer newly available therapy for late-onset Pompe disease

The first commercially available treatment in the United States for patients with late-onset Pompe disease was administered today (Wednesday, June 16) at the University of Florida.

17 Jun 2010

Help Conquer Cancer Project taps World Community Grid to discover structure of cancer-related proteins

Scientists have devised a way to automate and accelerate a manual, complex process that enables researchers to more easily discover the structure of cancer-related proteins, and, eventually, formulate cancer cures. This new, automated approach may also help the exploration of other diseases and food-related research.

15 Jun 2010

PPMD to fund two promising research initiatives in Duchenne muscular dystrophy

Patricia A. Furlong, Founding President and CEO of Parent Project Muscular Dystrophy (PPMD), the largest non-profit organization in the United States focused on finding a cure for Duchenne muscular dystrophy (Duchenne), announced today that PPMD will be funding two critical, promising research initiatives in Duchenne.

11 Jun 2010

Shenzhen Beike Biotechnology forms joint venture with SiriCell Technologies

Shenzhen Beike Biotechnology Co. Ltd., China's leading stem cell research and regenerative medicine company, announced the formation of Beike Holdings Limited a joint venture with Bangkok-based SiriCell Technologies, Inc. Beike Holdings, which is headquartered in Bangkok, Thailand, will focus primarily on the establishment of new international subsidiaries and the development of collaborative hospital partnerships with Southeast Asia's leading healthcare providers.

11 Jun 2010

Pharnext closes €4.8-million Series A funding round

Pharnext SAS, a biopharmaceutical company specializing in the development of innovative treatments based on Pleotherapy™ for severe neurological diseases, today announced that it had closed a €4.8-million Series A round of funding on June 3rd (corresponding to a company valuation of €45 million).

9 Jun 2010

$UC-MSCT improves symptoms, biochemical values in patients with severe refractory SLE: Study$

UC-MSCT improves symptoms, biochemical values in patients with severe refractory SLE: Study

A new study shows that umbilical cord mesenchymal stem cell transplant may improve symptoms and biochemical values in patients with severe refractory systemic lupus erythematosus.

8 Jun 2010

Medicare bidding program will lead to 'suicide bidding', says Pennsylvania Association of Medical Suppliers

Providers of home medical equipment and services across Pennsylvania 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 Pennsylvania's more than two million Medicare beneficiaries, and put hundreds of Pennsylvania's homecare providers out of business.

8 Jun 2010

Researchers underscore role of jumping genes in maintaining genetic diversity

Using high-throughput sequencing to map the locations of a common type of jumping gene within a person's entire genome, researchers at the University of Pennsylvania School of Medicine found extensive variation in these locations among the individuals they studied, further underscoring the role of these errant genes in maintaining genetic diversity.

4 Jun 2010

Highlights from June 2010 issue of American Journal of Pathology

Dr. Kanneboyina Nagaraju and colleagues at the Children's National Medical Center, Washington, DC demonstrate that affected muscle may directly contribute to inflammation in muscular dystrophy. Their report can be found in the June 2010 issue of The American Journal of Pathology.

26 May 2010

Repligen's RG2833 granted orphan drug designation for treatment of Friedreich's ataxia

Repligen Corporation announced today that the Office of Orphan Products Development of the Food and Drug Administration has granted orphan drug designation to RG2833, a selective histone deacetylase 3 inhibitor for the treatment of Friedreich's ataxia. Orphan drug designation qualifies Repligen to receive seven years of marketing exclusivity in the United States if the company is the first to obtain marketing approval for RG2833 for the treatment of Friedreich's ataxia.

24 May 2010

AMT reports positive preclinical data from cholesterol targeting AAV gene therapy study

Amsterdam Molecular Therapeutics a world leader in gene therapy, today reported positive preclinical data from a study using an AAV gene therapy product to lower cholesterol.

21 May 2010

Duchenne Muscular Dystrophy Awareness Week declared to honor work of PPMD

The Pennsylvania House of Representatives has passed a resolution declaring the week of May 24, 2010 as "Duchenne Muscular Dystrophy Awareness Week" to help honor the work of Parent Project Muscular Dystrophy, the largest non-profit organization in the United States focused on finding a cure for Duchenne muscular dystrophy.

15 May 2010

Repligen files IND for Phase 1 study of RG2833 selective HDAC-3 inhibitor for Friedreich's ataxia

Repligen Corporation announced today that it has filed an Investigational New Drug Application (IND) with the Food and Drug Administration (FDA) for a Phase 1 study of RG2833, a selective histone deacetylase 3 (HDAC-3) inhibitor. This is a double-blind, single ascending dose, Phase 1 study in healthy volunteers to evaluate the pharmacokinetic and safety profile of RG2833 in up to 40 subjects.

13 May 2010

Beike Biotechnology, Jiangsu government's CMC open world's largest stem cell management center

Beike Biotechnology Co. Ltd., one of the world's leading biotechnology companies focusing on adult stem cell therapies, and the Jiangsu government's China Medical City (CMC) opened China's state-of-the-art stem cell storage and processing facility today. The 20,000 square-meter Stem Cell Regenerative Medicine Industrial Project of National Bio-Industry Base (NBPD) houses China's first comprehensive regenerative medicine technology center and its largest international stem cell bank.

12 May 2010

Imperial College London and McGill University expand scientific collaboration

Neurological research and clinical care received a significant boost today as Imperial College London and McGill University of Montreal entered an agreement enabling them to work more closely together in this field.

11 May 2010

NIAMS, NINDS announce 5-year, $7.5M natural history study of Duchenne muscular dystrophy

The National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) and the National Institute of Neurological Disorders and Stroke (NINDS) are launching a five-year, $7.5 million natural history study of Duchenne muscular dystrophy (Duchenne), a degenerative genetically-linked neuromuscular disease. The study aims to validate non-invasive approaches to monitor the progression and treatment of Duchenne, and holds potential to facilitate the development of promising new therapies for people with the disease.

10 May 2010