SMA THERAPIES II AND BIOMARKERS

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Spinal muscular atrophy (SMA) is characterized by motor neuron loss and muscle atrophy, due to reduced levels of survival of motor neuron (SMN) protein from loss of function of the SMN1 gene. While SMN1 produces full-length SMN protein, a second gene, SMN2, produces only low levels of functional SMN protein. RG7916 (RO7034067) is an investigational, orally administered, centrally and peripherally distributed small molecule that modulates SMN2 pre-mRNA splicing towards the production of full-length SMN2 mRNA, resulting in an increase of SMN protein.

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