Amsterdam Molecular Therapeutics' AMT-080 granted EMEA Orphan Drug Designation
Amsterdam Molecular Therapeutics, a leader in the field of human gene therapy, announced today that the European Medicines Agency has granted Orphan Drug Designation to AMT's gene therapy product AMT-080 for the treatment of Duchenne muscular dystrophy.
posted in category: Medications/Drugs news tags: Duchenne Muscular Dystrophy Gene Therapy Muscular Dystrophy
Research shows exon-skipping drug may prove effective for treating Duchenne muscular dystrophy
An exon skipping PPMO has demonstrated dramatic effects in the prevention and treatment of severely affected, dystrophin and utrophin-deficient mice, preventing severe deterioration of the treated animals and extending their lifespan.
posted in category: Medications/Drugs news tags: Antisense Cell Duchenne Muscular Dystrophy Dystrophin Genetics Genomics Hospital Kyphosis Muscular Dystrophy Pathology Physiology Spine Utrophin Safety
Macrophages play important role in muscle regeneration
For scientists at the European Molecular Biology Laboratory (EMBL) in Monterotondo, Italy, what seemed like a disappointing result turned out to be an important discovery.
posted in category: Medications/Drugs news tags: Anti-Inflammatory Duchenne Muscular Dystrophy Macrophage Muscular Dystrophy Stroke Genes Heart Disease Sports injuries
Safeway raises funds to support neuromuscular disease medical research and therapies
Safeway Inc. announced today it has raised a record $10.2 million to support medical research and services for the millions of people living with neuromuscular diseases. The funds were raised during Safeway’s annual Muscular Dystrophy Association (MDA) campaign, a five-week endeavor to highlight the need to find cures and treatments for muscular dystrophy and related diseases, and presented during the Jerry Lewis MDA Telethon in Las Vegas Labor Day weekend.
posted in category: Diseases/Conditions tags: Duchenne Muscular Dystrophy Lou Gehrig's Disease Muscular Atrophy Muscular Dystrophy Neuromuscular Disease Spinal Muscular Atrophy Amyotrophic Lateral Sclerosis Grass Medical research
