Uses

Used for treatment of spinal muscular atrophy in pediatric and adult patients.

Do not use if

No specific contraindications established; avoid use in severe hypersensitivity.

Fever, diarrhea, rash, and upper respiratory tract infections may occur.

Use under specialist supervision with appropriate monitoring.

SMN2 splicing modifier that increases production of functional SMN protein.

Store at 20–25°C in the original bottle.