Muscular Dystrophy Animations

Potential Therapy For Congenital Muscular Dystrophy
Current research suggests laminin, a protein that helps cells stick together, may lead to enhanced muscle repair in muscular dystrophy. The related report by Rooney et al, "Laminin-111 restores regenerative capacity in a mouse model for alpha 7 integrin congenital myopathy," appears in the January 2009 issue of The American Journal of Pathology. Muscular dystrophy is a group of inherited genetic diseases that cause progressive muscle weakness.

EUR 7 Million Grants Boost Trophos Cholesterol-oxime Programs For ALS, MS And Other Neurodegenerative Diseases
Trophos SA, a clinical stage pharmaceutical company developing innovative therapeutics for indications with under-served needs in neurology and cardiology, announced that some EUR 7 million in funding has been awarded for two projects associated with its proprietary cholesterol-oxime class of compounds.

FYI From Human Genome Sciences: GSK Initiates Darapladib Phase 3 Development Program
Neuralstem, Inc. (NYSE Alternext US: CUR) announced this morning that it has filed an Investigational New Drug (IND) application with the U.S. Food and Drug Administration (FDA) to begin a clinical trial to treat amyotrophic lateral sclerosis (ALS or Lou Gehrig's disease). The Company is planning to treat ALS patients through spinal injections of its stem cells via its patented Human Neural Stem Cell technology.

MPs Put Spotlight On Specialist Care, UK
A cross party group of MPs and Peers have launched an Inquiry into access to NHS specialist care for people living with rare conditions. The Inquiry, being undertaken by the All Party Parliamentary Group for Muscular Dystrophy (APPG) will focus on access to specialist multi-disciplinary care; commissioning and funding; workforce planning and the role of care co-ordinators.

All Couples Should Be Offered Genetic Test For Spinal Muscular Atrophy, Says The ACMG
Carrier screening for spinal muscular atrophy (SMA) - a serious genetic disease affecting approximately 1 in 10,000 infants that causes progressive muscle weakness and death - should be made available to all families, according to a new practice guideline issued by the American College of Medical Genetics (ACMG). The statement appears in the November 2008 issue of Genetics in Medicine, the official peer-reviewed journal of the American College of Medical Genetics.

Genetic Change Extends Mouse Life, Points To Possible Treatment For ALS
There are many ways to die, but amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease, must be one of the worst. By the time a patient notices muscle weakness, the neurons that control the muscles have already begun dying, in an untreatable process that brings death within two to five years. In a series of experiments reported today (Dec.

Recent Developments At The Burnham Institute For Medical Research, Dec. 2008
Enzyme May Hold the Key to Muscular Dystrophy A mutation in the gene coding the protein dystrophin has long been known to be associated with muscular dystrophy, but the role the protein plays in the disease was unknown. Lorenzo Puri, M.D., Ph.D. and colleagues have discovered that the dystrophin mutation causes an increase in the amount of the histone deacetylase enzyme, HDAC2.

Maas Biolab SBIR Enters Phase 2 For Cyclosporin ALS Treatment
Maas Biolab has advanced to Phase 2 of its Small Business Innovation Research (SBIR) grant "Intrathecal Cyclosporin for the Treatment of Amyotrophic Lateral Sclerosis (ALS)." Jenny Karlsson, Ph.D., is Maas Director of Preclinical Development and Principal Investigator of the $2.1 million three-year grant from the National Institute of Neurological Disorders and Stroke (NINDS).

A Novel Human Stem Cell-based Model Of ALS Opens Doors For Rapid Drug Screening
Long thought of as mere bystanders, astrocytes are crucial for the survival and well-being of motor neurons, which control voluntary muscle movements. In fact, defective astrocytes can lay waste to motor neurons and are the main suspects in the muscle-wasting disease amyotrophic lateral sclerosis (ALS).

First Trial In Patients With A Potential Treatment Of The Incurable ALS Muscle Disease
Permission has been granted to start the first safety and tolerability trial on patients for a remedy for ALS. ALS is an incurable, paralyzing neurodegenerative disorder that strikes 5 persons in every 100,000. The disease commonly affects healthy people in the most active period of their lives - without warning. Researchers from VIB at the K.U.Leuven have previously shown the possibilities for the use of VEGF in the treatment of ALS through work in animal models.

A Model System To Elucidate The Origin Of Protein Misfolding Neurodegenerative Diseases
Amyotropic lateral sclerosis (ALS) is a neurodegenerative disease linked to the misfolding and aggregation of the dimers of the enzyme superoxide dismutase (SOD1). Other neurodegenerative disorders like Alzheimer's disease, Creutzfeld-Jakob disease and Parkinson's disease are also associated with protein misfolding and aggregation.

Growth Hormone Did Not Help ALS Patients, Study
A growth hormone that an earlier study had suggested might be effective, showed no benefit compared to placebo in treating patients with amyotrophic lateral sclerosis (ALS) or Lou Gehrig's disease, according to a new study performed by researchers in the United States. The research was conducted by lead author Dr Eric J Sorenson, of the Mayo Clinic in Rochester, Maine, USA, and colleagues, and was published in the 25 November issue of the journal Neurology.

Key Advance Toward Treatment For Most Common Adult Form Of Muscular Dystrophy - Journal Of The American Chemical Society
Scientists in New York are reporting a critical first step toward development of a long-sought drug to treat myotonic muscular dystrophy (MMD), the most common form of muscular dystrophy in adults. MMD affects about 1 in 8,000 people. Their findings appeared in the November 8 issue of ACS' weekly Journal of the American Chemical Society. In the study, Benjamin Miller and colleagues point out that MMD differs from typical hereditary diseases.

News From The American Chemical Society, Nov. 19, 2008
Microcapsules act as "roach motel" to kill harmful bacteria Researchers in New Mexico and Florida are reporting development of microscopic particles that act as chemical booby traps for bacteria. The traps attract and kill up to 95 percent of nearby bacteria, including microbes responsible for worrisome hospital-based infections. The scientists describe their discovery as micro-sized "roach motels" for harmful bacteria.

Neurons Derived From Embryonic Stem Cells Restore Muscle Function After Injury
Dalhousie Medical School researchers have discovered that embryonic stem cells may play a critical role in helping people with nerve damage and motor neuron diseases, such as amyotrophic lateral sclerosis (ALS), regain muscular strength. Motor neurons reside in the spinal cord and control limb movements by enabling muscles to contract. Diseases like ALS cause them to degenerate, resulting in muscle weakness, atrophy, and eventual paralysis.