Interim Data on Gene Therapy for SMA in Phase 1 Study Supports Safety, Potential Efficacy
AveXis releases the interim results of their Phase 1 clinical trial testing the gene therapy drug AVXS-101 for the treatment of SMA.
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SMA Patients Being Recruited for Phase 2 Study of Skeletal Muscle Therapy
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…
Therapy for Infantile-Onset SMA, Nusinersen, Shows Promise in Phase 3 Study
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…
2016 SMA Researcher Meeting Included Cutting-edge Science, Navigating FDA Process
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 to Put $2.5 Million Toward Research Efforts
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…
SMA Type 1 Treatment Candidate Granted FDA Breakthrough Therapy Designation
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…
SMA Muscle Breakdown Differs with Severity in Mouse Study, but Precedes Neuronal Death
In a study partly funded by Cure SMA, researchers from Ottawa Hospital Research Institute show that patterns of muscle breakdown in spinal muscular atrophy (SMA), occurring before any apparent neurodegeneration is taking place, differ depending on the severity of disease in mice. The report, “Differential induction…
Cure SMA Awards $50,000 Grant to Researcher Developing Potential Therapies
Cure SMA has awrded a $50,000 Drug Discovery Grant to Brigham and Women’s Hospital researcher Kevin Hodgetts, PhD, at the Lab for Drug Discovery in Neurodegeneration for his project “Development of a Drug to Increase SMN2 Transcription.” The grant Dr. Kevin HodgettsPhoto Credit: Cure…
Mutations That Cause SMA May Also Protect Against Infection, Hinting at Why They Continue to Thrive
Diminished levels of the survival of motor neuron (SMN) protein causes spinal muscular atrophy (SMA), and also disrupts a process called endocytosis — crucial for the transfer of nerve signals between cells. The findings, published in the journal PNAS in the study, “Decreased function of survival motor neuron…
Cure SMA Grants $50,000 to Researcher at Uniformed Services University of Health Sciences
Cure SMA awarded a $50,000 drug discovery grant to researcher Barrington Burnett of Uniformed Services University of the Health Sciences for the project “Slowing SMN degradation to treat SMA,” which aims to characterize and validate a new survival motor neuron (SMN) protein modulator for the treatment of spinal muscular atrophy (SMA). Drug discovery…
