Shots:

• The approval is based on P-II NURTURE study results assessing Spinraza in 300 infant patients who were genetically diagnosed with SMA and had not experienced any symptoms by the time of first dose aged 6 wks.
• The study resulted in early treatment allow 100% progressive gains in motor function- reduction in mortality in infants- 88% able to walk and safe and efficacious profile
• Spinraza (nusinersen) is antisense oligonucleotide (ASO) developed using Ionis’ antisense technology delivered using intrathecal injection directly to CSF targeted to treat SMA and is approved in 40 countries for SMA 

Ref: Biogen | Image: INA Pharmacy