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Cardiac stem cell treatment restores heart function damaged by Duchenne muscular dystrophy

Cardiac stem cell treatment restores heart function damaged by Duchenne muscular dystrophy

Researchers at the Cedars-Sinai Heart Institute have found that injections of cardiac stem cells might help reverse heart damage caused by Duchenne muscular dystrophy, potentially resulting in a longer life expectancy for patients with the chronic muscle-wasting disease. [More]
Isis receives $10 million from Biogen Idec to initiate IND-supporting studies of ISIS-BIIB3 Rx

Isis receives $10 million from Biogen Idec to initiate IND-supporting studies of ISIS-BIIB3 Rx

Isis Pharmaceuticals, Inc. announced today that it has earned a $10 million milestone payment from Biogen Idec related to the initiation of investigational new drug (IND)-supporting studies of ISIS-BIIB3Rx. ISIS-BIIB3Rx is a recently identified development candidate designed to inhibit an undisclosed target to treat a neurodegenerative disease. [More]
Wimpy antibody prevents more serious self-inflicted forms of kidney disease, researchers say

Wimpy antibody prevents more serious self-inflicted forms of kidney disease, researchers say

An antibody abundant in mice and previously thought to offer poor assistance in fighting against infection may actually play a key role in keeping immune responses in check and preventing more serious self-inflicted forms of kidney disease, researchers say. [More]
Study: Vitamin D deficiency more prevalent in neuromuscular conditions

Study: Vitamin D deficiency more prevalent in neuromuscular conditions

A study presented at the annual meeting of the American Association of Neuromuscular & Electrodiagnostic Medicine adds more credence to a growing awareness of the high prevalence of vitamin D deficiency in neuromuscular disease. [More]
Protein once seen as promising anti-cancer compound helps to stabilize neural circuits

Protein once seen as promising anti-cancer compound helps to stabilize neural circuits

Researchers at UC San Francisco (UCSF) have discovered that endostatin, a protein that once aroused intense interest as a possible cancer treatment, plays a key role in the stable functioning of the nervous system. [More]
Isis Pharmaceuticals begins ISIS-DMPK Rx Phase 1 study for treatment of Myotonic Dystrophy Type 1

Isis Pharmaceuticals begins ISIS-DMPK Rx Phase 1 study for treatment of Myotonic Dystrophy Type 1

Isis Pharmaceuticals, Inc. announced today that it has initiated a Phase 1 study for ISIS-DMPKRx. Isis earned a $14 million milestone payment from Biogen Idec associated with this achievement. [More]
Study: Sufficient copies of SMN1 gene extend survival in animals with spinal muscular atrophy

Study: Sufficient copies of SMN1 gene extend survival in animals with spinal muscular atrophy

To make up for insufficient amounts of SMN protein, the cause of the inherited neuromuscular disease spinal muscular atrophy (SMA), researchers have successfully delivered a replacement SMN1 gene directly to the spinal cords of animal models of SMA. [More]
Positive results from pivotal clinical trial of Trophos’ olesoxime in SMA patients to be presented at AAN 2014

Positive results from pivotal clinical trial of Trophos’ olesoxime in SMA patients to be presented at AAN 2014

Trophos and AFM-Telethon today announce that data from the pivotal clinical trial of Trophos’ lead product candidate olesoxime in spinal muscular atrophy (SMA) will be presented during the 66th American Academy of Neurology annual meeting to be held in Philadelphia, PA, USA, from April 26 to May 3 2014. [More]
Trophos' olesoxime shows beneficial effect on maintaining motor function in SMA patients

Trophos' olesoxime shows beneficial effect on maintaining motor function in SMA patients

Trophos today announces that top-line results from a pivotal clinical trial of its lead product candidate olesoxime in spinal muscular atrophy (SMA) show a beneficial effect on the maintenance of motor function in SMA patients. If approved, olesoxime could be the first treatment specifically developed for SMA patients. [More]
Scientists receive new awards from CIRM to advance revolutionary stem cell science in medicine

Scientists receive new awards from CIRM to advance revolutionary stem cell science in medicine

Scientists from UCLA's Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research have received new awards from the California Institute of Regenerative Medicine (CIRM), the state stem cell research agency, that will forward revolutionary stem cell science in medicine. [More]
ALS TDI forms research partnership with Neurimmune to advance potential treatments for ALS

ALS TDI forms research partnership with Neurimmune to advance potential treatments for ALS

The ALS Therapy Development Institute (ALS TDI), together with its wholly-owned subsidiary, Anelixis Therapeutics, announced today that it has formed a research partnership with Neurimmune to advance potential treatments for ALS, also known as Lou Gehrig's disease. [More]
Imaginative play with medically themed toys may improve recovery in chronically ill children

Imaginative play with medically themed toys may improve recovery in chronically ill children

New research finds that chronically ill children celebrate a successful recovery. It's through their imaginative play with medically themed toys. Laura Nabors, an associate professor of human services in the University of Cincinnati College of Education, Criminal Justice, and Human Services (CECH), will present new research on Tuesday, Nov. 5, at the American Public Health Association's (APHA) 141st Annual Meeting and Expo in Boston. [More]
Trophos completes efficacy study of olesoxime in patients with Spinal Muscular Atrophy

Trophos completes efficacy study of olesoxime in patients with Spinal Muscular Atrophy

Trophos SA, a clinical stage pharmaceutical company developing innovative therapeutics from discovery to clinical validation for indications with under-served needs in neurology and cardiology, has completed its efficacy study of olesoxime in the rare neurodegenerative condition Spinal Muscular Atrophy. [More]
Three Penn Medicine researchers awarded grants for neuromuscular disease research

Three Penn Medicine researchers awarded grants for neuromuscular disease research

The Muscular Dystrophy Association has awarded research grants totaling over $1,000,000 to three Penn Medicine researchers: James Shorter, PhD, associate professor of Biochemistry and Biophysics; Hansell Stedman, MD, associate professor of Surgery; and Lee Sweeney, PhD, director of the Center for Orphan Disease Research and Therapy. [More]
Quintiles and MDA partner to develop U.S. Neuromuscular Disease Registry

Quintiles and MDA partner to develop U.S. Neuromuscular Disease Registry

Quintiles and the Muscular Dystrophy Association (MDA) today announced a new partnership to develop and implement the U.S. Neuromuscular Disease Registry, a patient registry that will play an important role in determining effective treatments for people with muscular dystrophy and related muscle diseases. [More]
Isis Pharmaceuticals announces preliminary data from ISIS-SMNRx Phase 1 study in infants with SMA

Isis Pharmaceuticals announces preliminary data from ISIS-SMNRx Phase 1 study in infants with SMA

Isis Pharmaceuticals, Inc. announced today that follow-up preliminary data from a single dose, open-label Phase 1 study of ISIS-SMNRx in children with spinal muscular atrophy, show that most SMA children receiving the two highest doses of the drug (6 mg and 9 mg) continued to show improvements in muscle function tests up to 14 months after a single injection of the drug. [More]
Isis Pharmaceuticals starts ISIS-SMNRx Phase 2 study in infants with SMA

Isis Pharmaceuticals starts ISIS-SMNRx Phase 2 study in infants with SMA

Isis Pharmaceuticals, Inc. announced the initiation of a Phase 2 study of ISIS-SMNRx in infants with spinal muscular atrophy. SMA is a severe and rare genetic neuromuscular disease characterized by muscle atrophy and weakness and is the most common genetic cause of infant mortality. [More]
DART Therapeutics develops new class of therapy for Duchenne muscular dystrophy

DART Therapeutics develops new class of therapy for Duchenne muscular dystrophy

DART Therapeutics Inc., an innovative, new-model biotechnology firm focused on developing therapies for Duchenne muscular dystrophy, announced today that it is developing a SARM drug candidate obtained from Belgium-based Galapagos NV. [More]
Genetic testing services for rare neurological disorders launched by Athena Diagnostics

Genetic testing services for rare neurological disorders launched by Athena Diagnostics

Athena Diagnostics, a leader in neurological diagnostics, today announced the clinical availability of new genetic tests to aid the detection of several rare neurological disorders, including hereditary neuropathy, neuromuscular disease, epilepsy and certain movement disorders. The lab-developed tests are available through Athena Diagnostics, a business of Quest Diagnostics (NYSE:DGX), the world’s leading provider of diagnostic information services. [More]
Dementia diagnostic panel for treatable forms of cognitive impairment launched by Quest Diagnostics

Dementia diagnostic panel for treatable forms of cognitive impairment launched by Quest Diagnostics

Quest Diagnostics (NYSE: DGX), the world’s leading provider of diagnostic information services, announced the availability of the first clinical test panel for aiding the diagnosis of suspected dementia due to treatable forms of cognitive impairment. The test panel is believed to be the first commercial service from a clinical laboratory to combine several guideline-recommended tests for identifying secondary, treatable causes of dementia as a single blood test and report. [More]