Neuromuscular Disease News and Research

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MDA awards $14.1 million in new grants to advance treatments and cures for muscle diseases

The Muscular Dystrophy Association, which has invested almost $39 million in 2010 in worldwide research seeking treatments and cures for muscle diseases, today announced that it has awarded $14.1 million in new grants.

18 Aug 2010

Rxi Pharmaceuticals second-quarter net loss decreases to $2.1 million

RXi Pharmaceuticals Corporation, a recognized leader in RNAi-based therapeutic discovery and development, today reported its financial results for the second quarter ended June 30, 2010.

11 Aug 2010

Acceleron's ACE-031 protein therapeutic for DMD receives FDA Fast Track designation

Acceleron Pharma, Inc., 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 received FDA Fast Track designation for ACE-031 for the treatment of Duchenne Muscular Dystrophy (DMD), a fatal neuromuscular disease in which patients suffer from the progressive loss of muscle mass and strength. ACE-031 is an investigational protein therapeutic being developed to increase muscle mass and strength.

5 Aug 2010

Prosensa, GSK initiate additional programs for DMD

Prosensa, the Dutch biopharmaceutical company focusing on RNA modulating therapeutics, announces the initiation of two further programmes under its existing alliance with GSK covering novel RNA-based treatments for Duchenne Muscular Dystrophy (DMD). The initiation of these additional programs under the terms of the existing alliance agreement is a validation of both the potential of Prosensa's "exon skipping" platform and the ongoing relationship.

23 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

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

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

Acceleron Pharma commences Phase 2 clinical trial of ACE-031 protein therapeutic in DMD patients

Acceleron Pharma, Inc., 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 the initiation of a Phase 2 clinical trial with ACE-031 in patients with Duchenne Muscular Dystrophy (DMD), a fatal neuromuscular disease. ACE-031 is an investigational protein therapeutic that builds muscle and increases strength by inhibiting signaling of a cell surface receptor called the activin receptor type IIB (ActRIIB).

5 May 2010

Medication used to treat heart arrhythmias also reduces myotonic dystrophy: Researchers

A medication most often used to treat heart arrhythmias also reduces a central symptom of myotonic dystrophy, the most common type of muscular dystrophy in adults.

5 May 2010

Hospital for Special Care launches ALS Clinical Trials Unit at Neuromuscular Center

Hospital for Special Care announced today the launch of the region's first Amyotrophic Lateral Sclerosis (ALS) Clinical Trials Unit at its Neuromuscular Center. The new unit will be headed by Jinsy A. Andrews, M.D., a physician researcher recruited from Columbia University. Dr. Andrews, Director of Research & Clinical Trials Unit, Hospital for Special Care, specializes in neuromuscular research and will facilitate the hospital's first clinical trial for ALS upon her arrival.

23 Apr 2010

New therapeutic approach for Duchenne muscular dystrophy

Researchers from Universit- Laval's Faculty of Medicine and the CHUQ Research Center have proven that it is possible to repair the defective gene responsible for Duchenne muscular dystrophy.

16 Apr 2010

MDA awards over $21M in grants for advancing critical neuromuscular research

Forty-two medical researchers and their labs have been awarded more than $21 million in grants by the Muscular Dystrophy Association to advance critical neuromuscular research in 2010. Many of the grants are multi-year awards to be dispersed over the next three years.

15 Apr 2010

RXi Pharmaceuticals, miRagen Therapeutics enter into research collaboration

RXi Pharmaceuticals Corporation, a biopharmaceutical company pursuing the development and commercialization of proprietary therapeutics based on RNA interference, and miRagen Therapeutics, Inc., a biopharmaceutical company focused on improving patients' lives by developing innovative microRNA -based therapeutics for cardiovascular and muscle disease, announced today that they have entered into a research collaboration.

13 Apr 2010

Team of neurologists from the U.S. face Canadian neurologists in annual "Neurobowl"

In a competition modeled after TV quiz shows, a team of neurologists from the United States will face Canadian neurologists in the annual "Neurobowl®" on April 11.

12 Apr 2010

MDA, FFSHR award $200,000 grant for developing FSH dystrophy therapy

The Muscular Dystrophy Association (MDA), headquartered in Tucson, Ariz., and Friends of FSH Research (FFSHR), based in Kirkland, Wash., has jointly awarded a two-year, $200,000 grant to Dr. Joel Chamberlain, a research assistant professor of medical genetics at the University of Washington. The grant, equally funded by the two organizations, will enable the laboratory led by Dr. Chamberlain to study RNA interference as an investigative and therapeutic tool for facioscapulohumeral muscular dystrophy.

7 Apr 2010

Barrow Neurological Institute scientist receives grant to study myasthenia gravis

A scientist at Barrow Neurological Institute in Phoenix has been awarded a major grant from the Muscular Dystrophy Association to continue his groundbreaking work in the disease of myasthenia gravis.

6 Apr 2010

MDA awards 42 medical researchers and labs over $21M for critical neuromuscular research

Forty-two medical researchers and their labs have been awarded over $21 million by the Muscular Dystrophy Association to continue critical neuromuscular research. Many of the grants are multi-year awards to be dispersed over the next three years.

1 Apr 2010

Muscular dystrophy: $1M grant for study of erectile dysfunction drugs in improving muscle blood flow

A Cedars-Sinai Heart Institute cardiologist has been awarded a three-year, nearly $1 million grant from the Muscular Dystrophy Association (MDA) to study whether drugs used to treat erectile dysfunction could also be used to improve muscle blood flow and reduce fatigue in muscular dystrophy patients.

26 Mar 2010

Cardiologist receives grant to study impact of erectile dysfunction drugs on muscular dystrophy patients

25 Mar 2010