Muscular Dystrophy / ALS News From Medical News Today
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Diary Note: Nottingham Muscle Group Meeting, UK
Feb 23, 2010 12:00am
What Families affected by muscle disease in Nottingham and the East Midlands are meeting to hear how care and services in their region will be improved. Speaking at the meeting will be Kate Caston and Christine Richardson from the East Midlands NHS Specialised Commissioning Group......
William Hague Lends Muscle To Yorkshire And Humber Muscle Group, UK
Feb 21, 2010 1:00am
A group of campaigning muscle disease patients from Yorkshire and Humber are proud to announce the Rt. Hon. William Hague MP as their new patron. The Yorkshire and Humber Muscle Group asked Mr Hague, as MP for Richmond and a Rotherham-born local lad, to be patron of their organisation...
Sandra Gidley MP Flexes Political Muscle To Call For New Muscle Disease Care Advisor, UK
Feb 11, 2010 3:00am
Sandra Gidley MP is backing the fight for better muscle disease healthcare in Hampshire by calling on NHS bosses to fund specialist staff. Last month the South Central Muscle Group, a patients' forum for families in the region living with muscular dystrophy, met in Southampton where MP for Romsey...
Researchers Report That Tβ4 Increases Skeletal Muscle And May Have A Role In The Treatment Of Duchenne Muscular Dystrophy
Feb 8, 2010 3:00am
Regenerx Biopharmaceuticals, Inc. (NYSE Amex:RGN) announced today that a research team in Washington, D.C. has found that dystrophin-deficient Mdx mice, treated twice a week for six months with Tβ4, showed a significant increase in skeletal muscle regenerating fibers compared to untreated mice. No effects related to muscle function or fibrosis...
AVI BioPharma's Drug Candidate AVI-5038 Receives European Orphan Drug Designation For Duchenne Muscular Dystrophy
Feb 7, 2010 12:00am
AVI BioPharma, Inc. (NASDAQ: AVII), a developer of RNA-based drugs, announced that it received an orphan drug designation from the Committee for Orphan Medical Products of the European Medicines Agency (EMEA) for AVI-5038, a drug candidate being developed by AVI for the treatment of Duchenne Muscular Dystrophy (DMD). DMD is...
California Stem Cell And ALS Therapy Development Institute Extend Their Collaboration To Advance Potential Stem Cell Assisted Therapy For ALS
Feb 6, 2010 1:00am
California Stem Cell, Inc. (CSC) and ALS Therapy Development Institute (ALS TDI) are pleased to announce an extension and expansion of their collaboration aimed at advancing a potential stem cell therapy for ALS (amyotrophic lateral sclerosis)......
ALS Therapy Development Institute And Oxford BioMedica Begin Second Phase Of Collaboration To Develop Gene Therapies For ALS, UK
Feb 3, 2010 7:00pm
The ALS Therapy Development Institute and Oxford BioMedica announced today the extension of their collaboration following successful completion of the first phase......
Cytokinetics Announces Data From Phase I Multiple Dose Clinical Trial Of CK-2017357
Jan 28, 2010 1:00am
Cytokinetics, Incorporated (NASDAQ: CYTK) announced results from its Phase I, randomized, double-blind, placebo-controlled, multiple-dose clinical trial of oral CK-2017357. The primary objective of this clinical trial was to determine the safety and tolerability of CK-2017357 after multiple oral doses to steady state in healthy male volunteers......
University Of Florida Researchers Continue 'Extraordinary Measures' To Tackle Pompe Disease
Jan 26, 2010 12:00am
As scientists work to find new treatments for Pompe disease - the devastating genetic "villain" that drives the efforts of the main characters in the new film "Extraordinary Measures" - University of Florida researchers are hopeful that gene therapy will help patients in the late stages of the disease breathe...
Researchers Trace Effects Of Genetic Defect In Myotonic Muscular Dystrophy
Jan 25, 2010 2:00am
Research on the genetic defect that causes myotonic muscular dystrophy has revealed that the mutation disrupts an array of metabolic pathways in muscle cells through its effects on two key proteins......
Identification Of The Gene Responsible For A New Form Of Adult Muscular Dystrophy
Jan 25, 2010 12:00am
A study published in the online edition the American Journal of Human Genetics, allowed the first identification of a new form of adult onset muscular dystrophy. The research team led by Dr......
Neuralstem Announces First Patient Treated In ALS Stem Cell Trial
Jan 23, 2010 1:00am
Neuralstem, Inc. (NYSE Amex: CUR) announced that the first ALS patient was treated with its spinal cord stem cells yesterday at the Emory ALS Center at Emory University, in Atlanta, GA. A total of up to 18 patients is planned to be treated in this first U.S. clinical trial...
PTC Therapeutics Announces Additional Study Of Ataluren In Patients With Advanced Nonsense Mutation Duchenne/Becker Muscular Dystrophy
Jan 20, 2010 2:00am
PTC Therapeutics, Inc. (PTC) announced the initiation of an additional clinical trial of ataluren (PTC124®) in boys and young men with nonsense mutation Duchenne/Becker muscular dystrophy (nmDBMD) who have permanently lost the ability to walk independently. This trial is evaluating the best methods for measuring functional abilities in patients...
'Jekyll And Hyde' Cell May Hold Key To Muscular Dystrophy, Fibrosis Treatment: UBC Research
Jan 19, 2010 2:00am
A team of University of British Columbia researchers has identified fat-producing cells that possess "dual-personalities" and may further the development of treatments for muscle diseases such as muscular dystrophy and fibrosis. The team found a new type of fibro/adipogenic progenitors, or FAPs, that generate fatty fibrous tissues when transplanted into...
FDA Gives TCA Cellular Therapy Green Light To Proceed With First ALS Adult Stem Cell Trial Using Patient's Own Stem Cells
Jan 14, 2010 7:00pm
TCA Cellular Therapy, LLC (TCA-CT) announced that the U.S. Food and Drug Administration (FDA) has approved its adult stem cell protocol to conduct Phase I clinical trials to treat Amyotrophic Lateral Sclerosis (ALS or Lou Gehrig's disease)......
Investigators Identify Gene Mutations That Predispose Patients With Becker Muscular Dystrophy To Early Onset Cardiomyopathy
Jan 14, 2010 2:00am
Investigators in The Research Institute at Nationwide Children's Hospital have identified a link between specific modifications of the dystrophin gene and the age of cardiac disease onset in patients with Becker muscular dystrophy (BMD). This information could help clinicians provide early cardiac intervention for BMD patients based on genetic testing...
BioMarin Initiates Phase 1 Clinical Study Of BMN 195 For Duchenne Muscular Dystrophy
Jan 12, 2010 1:00am
BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced that the first subject has initiated treatment in the Phase 1 clinical study of BMN 195, a small molecule utrophin upregulator, for the treatment of Duchenne muscular dystrohpy (DMD). Initial top-line results are expected in the third quarter of 2010......
Muscular Dystrophy Association Renews Partnership With ALS Therapy Development Institute To Develop New Treatments For Lou Gehrig's Disease
Jan 8, 2010 1:00am
Buoyed by the extraordinary progress being made by the ALS Therapy Development Institute, the Muscular Dystrophy Association today announced a new milestone-driven grant of $2.5 million, adding to the $18 million MDA already has invested with ALS TDI -- the world's only non-profit research center focused exclusively on developing treatments...
New ALS Drug Slips Through Telling "Phase II" Clinical Trials
Jan 5, 2010 2:00am
A drug already used to treat symptoms of epilepsy has potential to slow the muscle weakening that comes with amyotrophic lateral sclerosis (ALS), scientists report after completing a Phase II clinical trial-an early, small-scale test to show if the drug works and continues to be safe......
Muscular Dystrophy Therapy Based On Tarantula-Venom To Be Advanced By UB Scientists' Biotech Company
Jan 4, 2010 1:00am
University at Buffalo biophysicists have found a protein in tarantula venom that shows promise as a potential therapy for muscular dystrophy (MD). They have formed a start-up biotech company in Buffalo -- Rose Pharmaceuticals -- to advance the drug to clinical trials......
Unusual Protein Modification Involved In Muscular Dystrophy, Cancer
Jan 4, 2010 12:00am
With the discovery of a new type of chemical modification on an important muscle protein, a University of Iowa study improves understanding of certain muscular dystrophies and could potentially lead to new treatments for the conditions. The findings, which appear in the Jan. 1, 2010, issue of the journal Science,...
Neuralstem Receives Approval To Commence First ALS Stem Cell Trial At Emory ALS Center
Dec 19, 2009 12:00am
Neuralstem, Inc. (NYSE Amex: CUR) announced that its Phase I trial to treat Amyotrophic Lateral Sclerosis (ALS or Lou Gehrig's disease) with its spinal cord stem cells has been approved by the Institutional Review Board (IRB) at Emory University in Atlanta, GA. The trial, which was approved by the FDA...
Prize4Life And The Jackson Laboratory Team Up To Fight ALS
Dec 12, 2009 12:00am
Prize4Life, an organization dedicated to finding a cure for amyotrophic lateral sclerosis (ALS, or Lou Gehrig's disease), is partnering with The Jackson Laboratory to help more researchers identify treatment candidates that increase the lifespan of mice that model ALS......
Tiny Molecule Slows Progression Of Lou Gehrig's Disease In Mice
Dec 11, 2009 4:00am
Researchers at UT Southwestern Medical Center have found that a molecule produced naturally by muscles in response to nerve damage can reduce symptoms and prolong life in a mouse model of amyotrophic lateral sclerosis (ALS). "We believe we can apply this research toward drug development," said Dr......
Knopp Neurosciences Reports Presentation Of Encouraging Clinical Trends In A Phase 2 Study Of KNS-760704 In ALS
Dec 11, 2009 2:00am
Knopp Neurosciences Inc. ("Knopp") announced the presentation of encouraging clinical results in a Phase 2 safety and tolerability study of KNS-760704 in amyotrophic lateral sclerosis ("ALS"). The results were presented at the 20th International Symposium on ALS/MND in Berlin, Germany, by Merit Cudkowicz, M.D......
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