Spinal muscular atrophy (SMA) patients in the European Union will soon have access to Spinraza (nusinersen), the first treatment approved for people with this disease and one able to treat almost all types of SMA. The European Commission announced late Thursday that it had approved Spinraza, marketed by…
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Continuous simulation training for family caregivers of children with spinal muscular atrophy (SMA) leads to improved assistance at home and better clinical outcomes, according to new research. The study, “An Evaluation of a Continuing Education Program for Family Caregivers of Ventilator-Dependent Children with Spinal Muscular Atrophy (SMA),” appeared in the…
Alberto Kornblihtt, PhD, professor and group leader at the Universidad de Buenos Aires in Argentina, has been awarded a $140,000 grant for a project examining genetic changes that affect how much SMN protein is produced from the SMN2 gene. The study may greatly impact the spinal muscular atrophy (SMA) research…
Cure SMA has awarded a $75,000 basic research grant to Dr. Stephen J. Kolb of Ohio State University (OSU) for a project that seeks a deeper understanding of motor neuron pathology in spinal muscular atrophy (SMA). The project, “Arrested Development or Neurodegeneration? An approach to understand developmental motor neuron pathology…
The U.S. Department of Health and Human Services; Advisory Committee on Heritable Disorders in Newborns and Children will review spinal muscular atrophy (SMA) for inclusion in the agency’s Recommended Uniform Screening Panel (RUSP). The application, prepared and submitted by a newborn screening working group involving staff and members of Cure…
Several experts are wondering how best to determine which spinal muscular atrophy (SMA) patients will benefit most from treatment with Spinraza (nusinersen), due both to its high cost and growing demand. Dr. Claudia A. Chiriboga, a neurology and pediatrics professor at Columbia University Medical Center, said Spinraza must be given every…
The U.S. Food and Drug Administration (FDA) has granted Cytokinetics’ investigational spinal muscular atrophy (SMA) treatment CK-2127107 Orphan Drug Designation. That gives it seven years of U.S. marketing exclusivity, tax credits for clinical research and a waiver from FDA user fees. CK-2127107, developed in partnership with Astellas Pharma, is…
As part of a $1.03 million basic research funding initiative, Cure SMA has awarded a $75,000 grant to University of Missouri researcher Chris Lorson, PhD, for his investigation into the role of astrocytes, a specific type of brain cells, in lower motor neuron susceptibility in spinal muscular atrophy…
Cure SMA has announced two additional grants to its current round of basic funding for research into spinal muscular atrophy (SMA) — a move that will boost the organization’s budget for grants to $1.03 million this year. The investment, which follows growing support from families, researchers, pharmaceutical firms and regulatory partners,…
In the roughly four months since the U.S. Food and Drug Administration (FDA) approved Spinraza (nusinersen) to treat all types of spinal muscular atrophy (SMA) in both children and adults, dozens of hospitals and clinics across the nation have begun dosing patients. Yet insurers are reluctant to pay for…
