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NINDS to Fund Biomarker Clinical Study for Spinal Muscular Atrophy

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SMA is associated with a mutation in chromosome 5 — loss of the survival of motor neuron 1 gene (SMN1) — and is considered the second most common autosomal recessive disease in the US, affecting 1 in 6000 live births. SMA Type 1 is one of the most common genetic causes of infantile death. Although the disease can be readily diagnosed, there are no approved treatments for SMA.

SMA is characterized by degeneration of motor neurons and progressive muscular atrophy. The clinical spectrum of SMA ranges from early infant death to normal adult life with only mild weakness.

Together with the Food and Drug Administration (FDA), the NINDS organized the Spinal Muscular Atrophy (SMA) Biomarker Qualification Workshop in May 2011 to discuss the current state of biomarker development in SMA. The discussions should provide information, but not limit, the choice of biomarkers proposed.

The NINDS intends to fund a single $1.8 million award in 2012 for a project lasting up to 3 years.

Source: NINDS

FOA: RFA-NS-12-004: Spinal Muscular Atrophy (SMA) Biomarker Study (U01)

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