Neuromuscular Disease News and Research

RSS

Muscular Dystrophy Association awards 44 research grants totaling $13.5 million

The Muscular Dystrophy Association today awarded 44 grants totaling $13.5 million to support new research to advance understanding of disease processes and to uncover new strategies for treating muscular dystrophies, ALS and other related neuromuscular diseases affecting more than a million Americans. The vital research funding was made possible by generous public support of the MDA Labor Day Telethon.

8 Feb 2011

CMD co-founder receives Prize4Life award for discovery of new ALS biomarker

Convergence Medical Devices (CMD) today announced that its co-founder, Dr. Seward Rutkove, Chief of the Division of Neuromuscular Disease at Beth Israel Deaconess Medical Center and Associate Professor of Neurology at Harvard Medical School, received Prize4Life's prestigious award for the discovery of a new biomarker for ALS (Amyotrophic Lateral Sclerosis), also known as Lou Gehrig's disease.

7 Feb 2011

Research shows nestin regulates formation of neuromuscular junction

In a study published in the Jan. 30, 2011, advance online edition of Nature Neuroscience, Salk Institute of Biological Studies investigators led by Kuo-Fen Lee, PhD., show that nestin has reason for being in a completely different cell type--muscle tissue.

30 Jan 2011

Genzyme to expand Geel facility to support growth of Myozyme and Lumizyme for Pompe disease

Genzyme Corp. today announced that it will build an additional manufacturing plant in Geel, Belgium, to support the long-term growth of Myozyme® and Lumizyme® for Pompe disease.

20 Jan 2011

GSK and Prosensa commence GSK2402968 Phase III study in DMD

GlaxoSmithKline and Prosensa today announced that the first patient has commenced treatment in the Phase III clinical study investigating GSK2402968, in ambulant boys with Duchenne Muscular Dystrophy (DMD), who have a dystrophin gene mutation amenable to an exon 51 skip (up to 13% of boys with DMD). Commencement of this study confirms previously announced plans to progress this asset into Phase III.

20 Jan 2011

Acceleron recieves $1.5 million MDA grant for ACE-031 clinical study in Duchenne Muscular Dystrophy

Acceleron Pharma, 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 has been awarded a $1.5 million grant from the Muscular Dystrophy Association to support clinical studies of ACE-031 in Duchenne Muscular Dystrophy, a disabling neuromuscular disease in which patients experience a progressive loss of muscle mass and strength.

6 Jan 2011

MDA awards Repligen $1.4M for research of new compound with therapeutic potential for SMA

The Muscular Dystrophy Association today announced it has awarded $1.4 million in new research funding to the biopharmaceutical firm Repligen of Waltham, Mass., to complete the preclinical work needed to begin human clinical trials of a promising therapeutic compound for spinal muscular atrophy (SMA).

17 Dec 2010

MDA supports Repligen with $1.4M grant for RG3039 Spinal Muscular Atrophy development program

Repligen Corporation today announced that the Company has received $1,400,000 in research funding from the Muscular Dystrophy Association ("MDA") to support the ongoing development of RG3039 for Spinal Muscular Atrophy ("SMA"). RG3039, our lead compound, is an inhibitor of an RNA processing enzyme which targets increased production of SMN, a protein of deficient levels in patients with SMA.

15 Dec 2010

SBUMC to rename and dedicate ALS clinic as Christopher Pendergast ALS Center of Excellence

For Chris Pendergast, every Christmas he has been alive to celebrate with his family for the past 17 years has been a gift unto itself. Indeed, in 1993 the then 44-year-old elementary school teacher from Miller Place on Long Island was diagnosed with amyotrophic lateral sclerosis - ALS - the always fatal degenerative neuromuscular disease commonly known as Lou Gehrig's Disease. He was told that he had, at best, three to five years to live.

15 Dec 2010

Pharnext receives French approval for Pleodrug Phase II clinical trial in Charcot-Marie Tooth disease type 1A

Pharnext SAS, a biopharmaceutical company specializing in the development of innovative treatments for severe neurological diseases and other major unmet medical needs, today announced that the French Agency for Healthcare Product Safety (AFSSAPS) has authorized a Phase II clinical trial with the first drug (a Pleodrug™) generated by the company's Pleotherapy™ technology platform in patients suffering from Charcot-Marie Tooth disease type 1A (CMT1A.

4 Dec 2010

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