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, is part of a strategic research plan aimed at advancing SMA research into its “next stage” by investigating SMA’s causes and biology, and ultimately finding a cure.
According to a press release issued May 8 by the Chicago-based nonprofit advocacy group, the plan is the collaborative effort of independent SMA experts, Cure SMA scientific advisory groups and a medicine and science committee. The grants are expected to help answer questions about survival motor neuron (SMN) protein — which is not produced sufficiently in people with SMA – and help identify other pathways and processes that are affected in such patients.
The basic research program has funded some of the most critical discoveries in the field of SMA. For instance, last month, Jocelyn Côté of Canada’s University of Ottawa received $140,000 to look for clues that could lead to new SMA treatment approaches. The grant will allow Côté and his team to investigate a newly discovered role that SMN plays in regulating protein production — thereby determining the potential consequences for SMA patients who lose this function.
Christine DiDonato of Chicago’s Northwestern Feinberg School of Medicine also received $140,000 from Cure SMA to examine how proteins in SMA skeletal muscle are wrongly regulated.DiDonato and her team will analyze normal versions of these misregulated proteins to determine their effect on the ability of SMA muscle to contract properly. Her findings could potentially help researchers understand how misregulation of proteins reduces force production and endurance in SMA muscle.
SMA, the leading genetic cause of death among U.S. infants, affects about one in 10,000 babies. About one in 50 Americans is a genetic carrier, and SMA can affect any race or gender.
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