Neuromuscular Disease News and Research

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Radical change to Alzheimer’s management

The first disease-modifying drugs are expected to launch from 2013, radically changing the way Alzheimer’s disease is treated. With the potential to successfully slow or halt the progression of Alzheimer’s, blockbuster revenues are beckoning for such pipeline drugs, claims independent market analyst, Datamonitor.

23 Nov 2010

Comprehensive guide to current practice of intraoperative neurophysiology

A new book entitled "Intraoperative Neurophysiologic Monitoring" has been published by Cambridge University Press, co-authored by Nationwide Children's Hospital Pediatric Neurologists Gloria M. Galloway, MD, and Khaled M. Zamel, MD; Marc R. Nuwer, MD, of UCLA; and Jaime R. Lopez, MD of Stanford University.

20 Nov 2010

28% of health care workers in 'work risk' category will not get flu vaccine

A new Consumer Reports Health poll reveals a disconcerting proportion of "at risk" groups who will not get the flu vaccine this year. Of most concern, only 40% of Americans in the "work risk" category—meaning those who care for young children and those who work in residential nursing homes, hospitals, and other health care environments—said they would definitely get the vaccine this year, which combines the seasonal and the 2009 H1N1 (swine) flus, while 28% said they would definitely not get the vaccine.

14 Oct 2010

Prosensa receives £7.5M milestone payment in GSK2402968 Phase IIa trial for DMD

Prosensa, the Dutch biopharmaceutical company focusing on RNA modulating therapeutics, has announced that it received a £7.5m milestone payment from GlaxoSmithKline (GSK) as a result of achieving a data milestone in its Phase IIa open label extension trial of GSK2402968 (PRO051), being developed to treat Duchenne Muscular Dystrophy (DMD) under its agreement with GSK. The milestone payment was based upon achievement of a successful safety review, with no serious safety signals observed.

13 Oct 2010

FDA designates NeuRx Diaphragm Pacing System as HUD device for ALS patients

Synapse Biomedical Inc. announces today that they have received the U.S. Food and Drug Administration (FDA) designation of the NeuRx Diaphragm Pacing System (DPS) as a Humanitarian Use Device (HUD) for amyotrophic lateral sclerosis (ALS) patients with a stimulatable diaphragm who are experiencing chronic hypoventilation and has now submitted for Humanitarian Device Exemption (HDE) approval.

11 Oct 2010

Immune reaction to dystrophin may contribute to muscle disease: Study

An immune reaction to dystrophin, the muscle protein that is defective in patients with Duchenne muscular dystrophy, may pose a new challenge to strengthening muscles of patients with this disease, suggests a new study appearing in the October 7, 2010, issue of The New England Journal of Medicine.

7 Oct 2010

ALS Society of Canada applauds Prime Minister's commitment to ensure benefits for veterans with ALS

The ALS Society of Canada applauds Prime Minister Stephen Harper's September 17 announcement on Global National Television regarding veterans with ALS: "I think I've been pretty clear that supporting our veterans is one of the government's highest priorities. We've been doing some internal reviews. We're aware of some concerns on how veterans who have ALS may be handled and I've certainly been insisting our officials look into how we can fix some people's problems. So you're going to see some action on that in the not-too-distant future."

21 Sep 2010

Study finds genetic change in chromosome 4 causes FSHD

The Muscular Dystrophy Association today heralds a landmark muscular dystrophy advance by an international study team of scientists and physicians from the Netherlands, United States, France and Spain. Led by MDA-grantee Silvère van der Maarel, Ph.D., at Leiden University Medical Center in the Netherlands, the collaborative study of more than 2,300 people found that two distinct genetic changes on chromosome 4 must be present to cause facioscapulohumeral muscular dystrophy (FSHD).

21 Aug 2010

Muscular Dystrophy Association awards $14.1M in new grants

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.

20 Aug 2010

FDA grants Acceleron's ACE-031 orphan designation for treatment of DMD

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 United States Food and Drug Administration (FDA) granted orphan designation for ACE-031 for the treatment of Duchenne Muscular Dystrophy (DMD), a fatal neuromuscular disease in which patients experience a progressive loss of muscle mass and strength. ACE-031 is an investigational protein therapeutic being developed to increase muscle mass and strength.

20 Aug 2010

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

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