Muscular Dystrophy News and Research

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The muscular dystrophies (MD) are a group of more than 30 genetic diseases characterized by progressive weakness and degeneration of the skeletal muscles that control movement. Some forms of MD are seen in infancy or childhood, while others may not appear until middle age or later. The disorders differ in terms of the distribution and extent of muscle weakness (some forms of MD also affect cardiac muscle), age of onset, rate of progression, and pattern of inheritance.

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

Amsterdam 2010 third quarter total expense increases from EUR 4.4 million to EUR 4.7 million

Amsterdam Molecular Therapeutics, a leader in the field of human gene therapy, today provides its non-audited business update in compliance with the EU transparency directive. This report summarizes material events and AMT's financial position for the third quarter of 2010.

18 Nov 2010

Researchers discover NG2+ cells may impact ALS disease progression

By tracking the fate of a group of immature cells that persist in the adult brain and spinal cord, Johns Hopkins researchers discovered in mice that these cells undergo dramatic changes in ALS, also known as Lou Gehrig's disease.

18 Nov 2010

New investigational drug is well tolerated in children with nmCF

PTC Therapeutics, Inc. today announced the publication of data from a Phase 2a clinical trial of ataluren in children with nonsense mutation cystic fibrosis (nmCF) in the American Journal of Respiratory and Critical Care Medicine.

15 Nov 2010

EuroNanoMed funds 8 projects with €8 Million for transnational research projects in nanomedicine

EuroNanoMed, a European funding initiative under the ERA-Net scheme of the European Commission aimed at advancing transnational research in Nanomedicine, announced today the projects that will be funded following its 2nd joint transnational call for collaborative research projects. Eight projects, involving 46 partners from 10 countries will be funded with €8 Million provided by EuroNanoMed partners. The research projects were chosen from 33 applications involving 178 applicant groups from 19 EU member and associated states/regions.

10 Nov 2010

Changes in Medicare policies may hamper supply of power wheelchairs

Will power wheelchairs continue to be available to Nevada's Medicare patients following a series of federal regulatory and policy changes?

9 Nov 2010

Insmed third quarter total revenues decrease to $1.8 million

Insmed Inc., a biopharmaceutical company, today reported results for the third quarter and nine-months ended September 30, 2010.

3 Nov 2010

Inefficient suppression of DUX4 gene causes facioscapulohumeral dystrophy: Study

An international team of researchers led by an investigator from Fred Hutchinson Cancer Research Center has made a second critical advance in determining the cause of a common form of muscular dystrophy known as facioscapulohumeral dystrophy, or FSHD.

29 Oct 2010

New gene therapy product reduces plasma cholesterol levels

Amsterdam Molecular Therapeutics (AMT) Holding N.V. a leader in the development of gene based therapies, today announced that its gene therapy product incorporating siRNA sequences into microRNA scaffolds to silence Apolipoprotein B100 (AAV-miApoB) was able to significantly lower plasma cholesterol levels in vivo over a period of 18 weeks. These preliminary results suggest that this approach could lead to a treatment for high cholesterol in humans

25 Oct 2010

PTC announces ataluren Phase 2b efficacy data at International Congress

PTC Therapeutics, Inc. today announced that final analyses of Phase 2b efficacy data suggest the investigational new drug ataluren slowed the loss of walking ability in patients with nonsense mutation dystrophinopathy, a disease continuum comprising Duchenne and Becker muscular dystrophy (nmDBMD). These data were presented at the International Congress of the World Muscle Society in Kumamoto, Japan and will be the basis of interactions with the U.S. Food and Drug Administration (FDA) and national regulatory authorities in Europe in the fourth quarter of 2010.

18 Oct 2010

Cellectis first half 2010 revenues increase 25%

Cellectis, the French genome engineering specialist, today published its consolidated financial statement for the half-year ending June 30, 2010: revenues increased 25% over H1 2009, expenditure increased 50% and the company ended the period with euro 38 million in cash reserves.

15 Oct 2010

Acceleron announces ACE-031 Phase 1b study results

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 preliminary results from a Phase 1b study to assess the safety, tolerability and pharmacodynamic (PD) activity of ACE-031 following multiple ascending doses in healthy postmenopausal volunteers.

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

Kennedy Krieger Institute launches phase II clinical trial to investigate Revatio for DMD treatment

Kennedy Krieger Institute announced today the launch of a first‐of‐its‐kind, phase II clinical trial to investigate a treatment for heart disease in individuals with Duchenne muscular dystrophy (DMD).

13 Oct 2010

Scientists identify molecular mechanism of muscle stem cell differentiation

Under normal circumstances, adult stem cells reside in muscle tissue, where they can differentiate into a number of different cell types. After an injury (or even a tough workout), muscles are inflamed as cells and molecules flood the area to control damage and begin repairs. When called upon to replace muscle tissue damaged by injury or genetic disease, some muscle stem cells differentiate, becoming new muscle cells, while others make more stem cells. At Sanford-Burnham Medical Research Institute (Sanford-Burnham), a team of scientists led by Pier Lorenzo Puri, M.D., Ph.D., recently uncovered the molecular messengers that translate inflammatory signals into the genetic changes that tell muscle stem cells to differentiate.

11 Oct 2010

Two-day symposium marks an extraordinary decade for Brown Institute for Brain Science

Just as intense concentration allows a sharp mind to perform at its peak, a research institute that concentrates pre-eminent scientists from 11 academic departments can push back the frontiers of knowledge in a complex research area: the human brain.

8 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

Patients with muscular dystrophy mount immune response to dystophin protein prior to gene therapy: Study

A clinical trial designed to replace the genetic defect causing the most common form of muscular dystrophy has uncovered an unexpected aspect of the disease. The trial, based on therapy designed by scientists at the University of North Carolina at Chapel Hill School of Medicine, showed that some patients mount an immune response to the dystrophin protein even before they have received the gene therapy.

7 Oct 2010

Hundreds of experts including Nobel laureates say Medicare's bidding system for durable medical equipment will fail

Medicare's bidding program for durable medical equipment will fail, say 166 leading experts on auctions and competitive bidding systems.

1 Oct 2010

NIH awards three grants to explore novel treatment strategies for muscular dystrophy

Three grants totaling more than $4.5 million, from agencies of the National Institutes of Health, will be used to explore novel treatment strategies for muscular dystrophy.

30 Sep 2010