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Genentech's Risdiplam Meets Primary Goal In Type 1 Spinal Muscular Atrophy Trial

Genentech, a member of the Roche Group (RHHBY), said that its Risdiplam met primary endpoint in pivotal firefish trial in infants with type 1 spinal muscular atrophy.

Risdiplam demonstrated statistically significant and medically meaningful motor milestone improvement in infants with Type 1 spinal muscular atrophy.

The primary outcome measure of the study was the proportion of infants sitting without support for at least five seconds at 12-months of treatment, assessed by the Gross Motor Scale of the Bayley Scales of Infant and Toddler Development - Third Edition.

Spinal muscular atrophy or SMA is a severe, inherited, progressive neuromuscular disease that causes devastating muscle atrophy and disease-related complications. It is the most common genetic cause of infant mortality and one of the most common rare diseases, affecting approximately one in 11,000 babies.

Spinal muscular atrophy leads to the progressive loss of nerve cells in the spinal cord that control muscle movement. An individual's physical strength and their ability to walk, eat or breathe can be significantly diminished or lost, depending on the type of SMA.

SMA is caused by a mutation in the survival motor neuron-1 gene that results in a deficiency of SMN protein. SMN protein is found throughout the body and increasing evidence suggests SMA is a multi-system disorder and the loss of SMN protein may affect many tissues and cells, which can stop the body from functioning.

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