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…
News
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…
Blocking a protein receptor called m2R may prevent motor neuron death, leading to new therapies for spinal muscular atrophy (SMA), according to a study. The research, “Decreased microRNA Levels Lead To Deleterious Increases In Neuronal M2 Muscarinic Receptors In Spinal Muscular Atrophy Models,” was published in the online journal…
The nonprofit advocacy group Cure SMA has awarded Christine DiDonato a $140,000 grant to study how specific proteins reduce force production and endurance in muscles affected by spinal muscular atrophy (SMA). SMA is a childhood disease that occurs in one of 10,000 births. It is characterized by degeneration of the motor neurons in the…
A crucial two-hour meeting between AveXis executives and officials of the U.S. Food and Drug Administration (FDA) will determine the next step for AVXS-101, a spinal muscular atrophy (SMA) treatment that has shown remarkable promise in a Phase 1 trial of babies with SMA type 1 — the most severe form of this muscle-damaging disease.
A new test that can detect the risk of having children with spinal muscular atrophy (SMA) will be commercialized in a joint effort by the two companies, SYNLAB and Counsyl. SYNLAB will offer Counsyl’s proprietary Expanded Carrier Screening (ECS) test under the tradename preconGEN. The test will be offered…
