Screening for spinal muscular atrophy (SMA) in high-risk groups could greatly reduce the impact of the disease, according to a new article published by researchers in Ottawa. The report, “Opening the window: The case for carrier and perinatal screening for spinal muscular atrophy,” was published in the journal Neuromuscular Disorders.
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Cure SMA-funded researcher Dr. Christian L. Lorson and his colleagues have published a paper in the journal Molecular Therapy describing a promising therapeutic prospect in a mouse model with severe spinal muscular atrophy (SMA). The article, “Optimization of Morpholino Antisense Oligonucleotides Targeting the Intronic Repressor Element1 in Spinal…
Cure SMA has created a series of educational modules to address the topics, issues, and questions that are critical as the development of new drugs moves forward. The organization says a new stage has been reached in the efforts for U.S. FDA-approved treatments for spinal muscular atrophy (SMA),…
August, proclaimed SMA Awareness Month, is when advocacy and research organizations like CureSMA and the SMA Foundation and supporters seek to raise public consciousness of the genetic disorder spinal muscular atrophy (SMA). While the SMA support community works year-round to raise awareness of and funds for…
AveXis releases the interim results of their Phase 1 clinical trial testing the gene therapy drug AVXS-101 for the treatment of SMA.
A Phase 2 clinical trial investigating a potential treatment, CK-2127107, in people with spinal muscular atrophy (SMA), ages 12 years and older, is currently recruiting patients in the U.S. with varying degrees of disease severity (Types 2, 3, or 4). CK-2127107 is an investigational drug candidate designed for skeletal muscle and other debilitating neuromuscular…
Biogen and Ionis Pharmaceuticals recently reported that nusinersen, an antisense drug designed to treat spinal muscular atrophy (SMA), ably met the primary endpoint set for an interim analysis of its Phase 3 ENDEAR clinical trial. That study, evaluating nusinersen as a treatment for infantile-onset (consistent with Type 1) SMA, is now closing and the program expanding…
The 2016 SMA Researcher Meeting, the largest gathering specifically dedicated to spinal muscular atrophy (SMA), set a record with 350 attendees who discussed cutting-edge research and the future of SMA patients’ needs. The goal of the annual event, held this year in Anaheim, California, is to create an open forum for the…
Cure SMA, an organization dedicated to the treatment and cure of Spinal Muscular Atrophy (SMA), will provide $2.5 million over the next 12 months for research and to ensure the development of therapies for all ages, types, and stages of SMA. The announcement was made during the organization’s 2016 Annual SMA…
The U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to AVXS-101, AveXis’ lead development candidate to treat spinal muscular atrophy (SMA) Type 1 in pediatric patients. AVXS-101 is a proprietary gene therapy candidate of a one-time, intravenous treatment for SMA Type 1, and, according to AveXis, is the…
