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.

AMT receives CHMP opinion on Glybera Marketing Authorisation Application

Amsterdam Molecular Therapeutics, a leader in the field of human gene therapy, today announced that it has received an opinion on its Marketing Authorisation Application (MAA) for Glybera as a potential therapy for Lipoprotein Lipase Deficiency.

24 Jun 2011

FDA grants Fast Track designation for Repligen's RG3039 to treat Spinal Muscular Atrophy

Repligen Corporation announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for RG3039, a potential treatment for Spinal Muscular Atrophy.

23 Jun 2011

Reduced levels of VAPB protein may play central role in causing ALS

An international team of scientists led by researchers at the University of California, San Diego School of Medicine have used induced pluripotent stem cells (iPSCs) derived from patients with amyotrophic lateral sclerosis (ALS) to reveal for the first time how reduced levels of a specific protein may play a central role in causing at least one inherited form of the disease.

22 Jun 2011

Miracle cures on the horizon for genetic disease

In a latest break through, scientists have found a new technique to alter the genetic code behind hundreds of diseases and this was hailed by U.S. scientists as a “miracle of modern medicine.” Genes responsible for devastating diseases can be corrected by controlling faulty messenger cells that are central to the body's protein production, researchers said Wednesday.

17 Jun 2011

Guidelines for pediatric use of polysomnography for sleep-disordered breathing before tonsillectomy

A multidisciplinary clinical practice guideline, "Polysomnography for Sleep-Disordered Breathing Prior to Tonsillectomy in Children" will be published as a supplement to the July issue of Otolaryngology-Head and Neck Surgery.

15 Jun 2011

New understanding of muscle growth and repair

Revealing another part of the story of muscle development, Johns Hopkins researchers have shown how the cytoskeleton from one muscle cell builds finger-like projections that invade into another muscle cell’s territory, eventually forcing the cells to combine.

5 Jun 2011

Insight into understanding of normal muscle growth

Revealing another part of the story of muscle development, Johns Hopkins researchers have shown how the cytoskeleton from one muscle cell builds finger-like projections that invade into another muscle cell's territory, eventually forcing the cells to combine.

4 Jun 2011

Consumer groups, market experts support repeal of Medicare bidding program

Providers of home medical equipment and services in Oregon support H.R. 1041, a bipartisan bill in Congress that would repeal the controversial Medicare "competitive" bidding program for certain medically required equipment and services used at home.

4 Jun 2011

Salk researchers successfully edit diseased gene in patient-specific induced pluripotent stem cells

In principle, genetic engineering is simple, but in practice, replacing a faulty gene with a healthy copy is anything but. Using mutated versions of the lamin A gene as an example to demonstrate the versatility of their virus-based approach, researchers at the Salk Institute for Biological Studies successfully edited a diseased gene in patient-specific induced pluripotent stem cells as well as adult stem cells.

21 May 2011

AMT presents long-term efficacy study of Glybera in treatment of lipoprotein lipase deficiency

Amsterdam Molecular Therapeutics, a leader in the field of human gene therapy, announced today results from a long-term efficacy study of Glybera (alipogene tiparvovec) that showed improved chylomicron metabolism could be used as a biomarker for increased lipoprotein lipase (LPL) activity in those patients missing the gene that produces this protein.

20 May 2011

Stem cells from kidneys - World first from Australian scientists

According to Australian scientists, their latest stem cell research breakthrough will bring new treatments for a growing epidemic of chronic kidney disease. They are the first in the world to generate a type of stem cell from human kidney cells. The Melbourne scientists are hoping their discovery will have far-reaching effects in treating the disease.

19 May 2011

Novel gene associated with heart development and aging process

Researchers at the University of Ottawa Heart Institute (UOHI) have identified a novel gene in the nucleus of muscle and brain cells that affects heart development and the aging process. Their investigation brings the promise of new treatments for an old, failing heart.

18 May 2011

Losartan therapy improves muscle injury regeneration and prevents disuse atrophy

Using geriatric mice, a Johns Hopkins research team has shown that losartan, a commonly used blood pressure drug, not only improves regeneration of injured muscle but also protects against its wasting away from inactivity.

12 May 2011

Being mildly overweight benefits ALS patients

Patients with amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease, may be an exception to the rule that being overweight is a health hazard. In a retrospective study of over 400 ALS patients, Massachusetts General Hospital researchers found that those who were mildly obese survived longer than patients who were normal weight, underweight or even overweight.

12 May 2011

UCSF scientists receive CIRM grants to advance degenerative muscle disease research

Three UCSF scientists have received grants from the California Institute for Regenerative Medicine to advance their investigations of treatment strategies for degenerative muscle diseases, such as muscular dystrophy, and heart disease, and to determine why human embryonic stem cells are susceptible to forming tumors.

10 May 2011

Scripps Research receives CIRM grant for new research in stem cell biology and disease origins

Stem cells have huge potential in medicine because they have the ability to change or differentiate into many different cell types potentially providing a source of cells to replace those that have been permanently lost by a patient.

6 May 2011

Researchers discover two mutations responsible for neurological condition

Mayo Clinic researchers have discovered two mutations responsible for a devastating neurological condition they first identified 15 years ago. The researchers say their study -- appearing in Nature Genetics -- has revealed a new neural pathway that may help understand a variety of similar conditions.

2 May 2011

Abnormal connections created by central nervous system linked to persistence of chronic pain

Chronic pain is an epidemic. One in four Americans suffers from chronic pain due to disease—including cancer and HIV—and the medications used to treat those diseases. One reason for the persistence of chronic pain may be that the patient's central nervous system creates abnormal connections or improves connections that shouldn't be strengthened, explains Susan G. Dorsey, PhD, RN, co-director of the School of Nursing's Center of Excellence in Disorders of Neuroregulatory Function.

30 Apr 2011

Scientists develop novel genetic model of premature aging disorders

Working with a group of national and international researchers, scientists from the Florida campus of The Scripps Research Institute have developed a new genetic model of premature aging disorders that could shed light on these rare conditions in humans and provide a novel platform for large-scale screening of compounds to combat these and other age-related diseases.

29 Apr 2011

Tweaking gene in satellite cells may increase lifespan of people with muscular dystrophy

Researchers have long questioned why patients with Duchenne muscular dystrophy (DMD) tend to manage well through childhood and adolescence, yet succumb to their disease in early adulthood, or why elderly people who lose muscle strength following bed rest find it difficult or impossible to regain.

18 Apr 2011