Muscular Dystrophy / ALS News From Medical News Today Latest Health News and Medical News posted throughout the day, every day.
Is Prenatal Screening For Rare Diseases Like Spinal Muscular Atrophy Too Costly?
Spinal Muscular Atrophy (SMA) is one of many serious disorders for which prenatal testing is available. SMA affects approximately 1 in 10,000 live births and is the leading genetic cause of infant mortality and the second most common autosomal recessive disorder, after cystic fibrosis...
ISIS Initiates Phase 1 Clinical Trial Of ISIS-SOD1Rx In Patients With ALS
Isis Pharmaceuticals, Inc. (Nasdaq: ISIS) announced that it has initiated a Phase 1 study of ISIS-SOD1Rx in patients with an inherited, aggressive form of Lou Gehrig's disease also known as familial amyotrophic lateral sclerosis (ALS). Approximately 20 percent of all familial ALS cases are caused by a mutant form of superoxide dismutase, or SOD1...
Diary Note: Nottingham Muscle Group Meeting, UK
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
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 and are pleased that he has accepted this post...
Clive Svendsen Of Cedars-Sinai To Receive ALS Research Award From American Academy Of Neurology
Clive Svendsen, Ph.D., director of the Cedars-Sinai Regenerative Medicine Institute, is receiving the American Academy of Neurology Sheila Essey Award for his research on ALS (amyothrophic lateral sclerosis). ALS, often referred to as Lou Gehrig's disease, is a progressive neurodegenerative disease in which nerve cells called motor neurons degenerate in the brain and spinal cord...
Sandra Gidley MP Flexes Political Muscle To Call For New Muscle Disease Care Advisor, UK
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 Sandra Gidley spoke to them about making their voices heard by politicians...
Researchers Report That TГЋВІ4 Increases Skeletal Muscle And May Have A Role In The Treatment Of Duchenne Muscular Dystrophy
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 TГЋВІ4, showed a significant increase in skeletal muscle regenerating fibers compared to untreated mice. No effects related to muscle function or fibrosis and no adverse reactions were observed in the mice...
AVI BioPharma's Drug Candidate AVI-5038 Receives European Orphan Drug Designation For Duchenne Muscular Dystrophy
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 a genetic muscle-wasting disease caused by failure to produce dystrophin...
California Stem Cell And ALS Therapy Development Institute Extend Their Collaboration To Advance Potential Stem Cell Assisted Therapy For ALS
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
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
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
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 on their own...
Researchers Trace Effects Of Genetic Defect In Myotonic Muscular Dystrophy
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
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
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 to evaluate human neural stem cells for the treatment of ALS (Amyotrophic Lateral Sclerosis, or Lou Gehrig's disease)...
PTC Therapeutics Announces Additional Study Of Ataluren In Patients With Advanced Nonsense Mutation Duchenne/Becker Muscular Dystrophy
PTC Therapeutics, Inc. (PTC) announced the initiation of an additional clinical trial of ataluren (PTC124В®) 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 who have lost independent mobility...
'Jekyll And Hyde' Cell May Hold Key To Muscular Dystrophy, Fibrosis Treatment: UBC Research
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 damaged muscles in mice...
FDA Gives TCA Cellular Therapy Green Light To Proceed With First ALS Adult Stem Cell Trial Using Patient's Own Stem Cells
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
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 results performed on a blood sample...
BioMarin Initiates Phase 1 Clinical Study Of BMN 195 For Duchenne Muscular Dystrophy
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
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 for amyotrophic lateral sclerosis (ALS)...
New ALS Drug Slips Through Telling "Phase II" Clinical Trials
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
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
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, may also have implications for detecting metastasizing cancer cells...
Neuralstem Receives Approval To Commence First ALS Stem Cell Trial At Emory ALS Center
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 in September, will take place at the Emory ALS Center, under the direction of Dr...
Worlds Best Pharmacy
canadian meds shop
Why is your product so cheap? How do you ship orders? We accept : Visa, MasterCard, AmEx, Diners and JCB cards. |
