Duchenne muscular dystrophy (DMD) is a progressive muscle disorder that causes the loss of both muscle function and independence. DMD is perhaps the most prevalent of the muscular dystrophies and is the most common lethal genetic disorder diagnosed during childhood today. Each year, approximately 20,000 children worldwide are born with DMD (one of every 3,500 male children).
Amsterdam Molecular Therapeutics, a leader in the field of human gene therapy, announced today that the Duchenne Parent Project, based in the Netherlands, has awarded AMT a grant of EUR 145,000 to support the development of AMT-080, AMT's gene therapy for Duchenne Muscular Dystrophy.
Amsterdam Molecular Therapeutics, a leader in the field of human gene therapy, today reported its results for the year to December 31, 2010.
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.
Everyone knows chocolate is critical to a happy Valentine's Day. Now scientists are one step closer to knowing what makes a heart happy the rest of the year.
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.
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.
Scientists at Royal Holloway, University of London have reported encouraging results in a new gene-based therapy for Duchenne Muscular dystrophy (DMD) which at present has no known cure and affects one in 3,000 young boys.
A novel potential therapy based on a natural human protein significantly slows muscle damage and improves function in mice who have the same genetic mutation as boys with the most common form of muscular dystrophy, according to a paper published online Dec. 27 in the Proceedings of the National Academy of Sciences.
Children with Duchenne muscular dystrophy face a future of rapidly weakening muscles, which usually leads to death by respiratory or cardiac failure before their 30th birthday. While researchers are hopeful that gene therapy could eventually evolve into an effective treatment, few have targeted the heart of the problem as much as Dongsheng Duan, PhD.
Amsterdam Molecular Therapeutics Holding N.V., a leader in the development of gene-based therapies, today announced that it has amended and restated its licensing agreement with Amgen for gene therapy applications incorporating the GDNF (glial cell derived neurotrophic factor) gene, to which Amgen holds rights. Financial terms were not disclosed.
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.
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
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.
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.
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.
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.
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).
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.
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.
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.
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