Researchers from The Jackson Laboratory in Maine, in collaboration with Isis Pharmaceuticals, tested a novel therapeutic candidate (antisense oligonucleotides) in genetically engineered mice with spinal muscular atrophy (SMA), observing it could restore some functionality of motor neurons. The study entitled “Systemic, postsymptomatic antisense oligonucleotide rescues motor…
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AveXis, Inc. has recently presented results from its ongoing Phase 1/2 trial of AVXS-101 for the treatment of spinal muscular atrophy (SMA) Type 1 during the International Congress of the World Muscle Society in Brighton, United Kingdom. Lead investigator Dr. Jerry R. Mendell, director of the Center for Gene…
Children with spinal muscular atrophy (SMA) have weaker muscles than children who are not affected by muscle myopathies and new results from a study conducted at Washington University School of Medicine in St. Louis, Missouri, indicate this muscle weakness may be due to differences in muscle thickness. Researchers used ultrasound…
The Ensuring Access to Clinical Trials Act of 2015 (EACT) has been passed by the U.S. House of Representatives, and having already been passed the Senate in July, the bill will now go to President Obama to be signed into law. The EACT bill authorizes individuals to receive up to…
Cure SMA, an organization dedicated to the treatment and cure of spinal muscular atrophy (SMA), recently revealed novel studies presented during its annual researcher meeting. SMA is a genetically-determined neuromuscular disorder characterized by loss of specific spinal cord neurons, which causes weakness of the limbs and muscle degeneration. This leads to loss of voluntary muscle…
A team of researchers from Harvard University and the Karolinska Institute in Sweden unveiled the cellular pathway leading to the specific death of motor neurons in spinal muscular atrophy (SMA). The study entitled “Genome-wide RNA-Seq of Human Motor Neurons Implicates Selective ER Stress Activation in Spinal Muscular…
Cure SMA, one of the country’s leading not-for-profit organizations committed to find a cure for spinal muscular atrophy and improve the lives of those afflicted by the disease, has been conducting its series of SMA Researcher Meetings which began earlier this year in June and will end in December. The organization regularly updates its…
Buffalo, New York-based not-for-profit organization Spinal Muscular Atrophy Research Team (SMART) recently announced it awarded $50,000 to Cure SMA to help fund ongoing research for a cure for this debilitating disease. Since its founding in 2009, SMART has selected promising scientists and institutions to receive research grants to further SMA…
Dallas-based gene therapy platform company AveXis Inc., a leader in developing treatments for rare and life-threatening neurological genetic diseases, has announced completion of a $65 million Class D common stock financing. The proceeds will be used to advance the company’s ongoing Spinal Muscular Atrophy (SMA) research and clinical programs and…
When a protein is missing in a cell, as in the case of spinal muscular atrophy (SMA) where survival motor neuron (SMN) is missing, a variety of methods can be used to restore function to cells lacking the protein and treat the disease. Direct delivery of the protein or a…
