First drug approved for treatment of spinal muscular atrophy

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FDA approved Spinraza (nusinersen), the first drug to treat spinal muscular atrophy (SMA), a rare and often fatal genetic disease affecting muscle strength and movement.

SMA is a genetic disease that causes weakness and muscle wasting because of the loss of lower motor neurons controlling movement. SMA is classified in many types based on age of onset, severity of muscle weakness and symptoms. Spinraza is an injection administered into the fluid surrounding the spinal cord, approved for use across the range of spinal muscular atrophy patients.

The efficacy of Spinraza was demonstrated in a clinical trial including 121 patients (aged < 7 mo at the time of 1st dose) with infantile-onset SMA diagnosed before 6 months of age. Patients were randomized to receive an injection of Spinraza, into the fluid surrounding the spinal cord, or undergo a mock procedure without drug injection (a skin prick). In an interim analysis 40% of patients treated with Spinraza achieved improvement in motor milestones (head control, sitting, ability to kick in supine position, rolling, crawling, standing and walking), whereas none of the control patients did.

The findings of additional open-label uncontrolled clinical studies conducted in symptomatic patients (aged 30 days to 15 yrs at the time of 1st dose) and presymptomatic patients (aged 8 days to 42 days at the time of 1st dose) support the clinical efficacy demonstrated in the controlled clinical trial in infantile-onset patients.

The most common side effects reported in patients...

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