mySMAteam

Currently, there are three FDA-approved treatments for SMA:

• Nusinersen (Spinraza) - given by spinal injection, approved for all ages
• Risdiplam (Evrysdi) - oral medication available for all ages
• Onasemnogene abeparvovec-xioi (Zolgensma) - currently only approved for children under age 2

Both Spinraza and Evrysdi work Show Full Answer

Currently, there are three FDA-approved treatments for SMA:

• Nusinersen (Spinraza) - given by spinal injection, approved for all ages
• Risdiplam (Evrysdi) - oral medication available for all ages
• Onasemnogene abeparvovec-xioi (Zolgensma) - currently only approved for children under age 2

Both Spinraza and Evrysdi work by increasing SMN protein production from the SMN2 gene and are available treatment options for adults. While Zolgensma is currently only approved for young children, researchers are studying its potential use in adults. Early research suggests the viral delivery method used in Zolgensma might be suitable for adult patients regardless of their age, SMA type, walking ability, or ventilatory status. However, this is still being investigated through clinical trials and is not yet an approved treatment option for adults.

 AI-generated responses are not a substitute for medical advice. These services are not intended to diagnose, treat, or cure any condition or disease. Always ask your doctor about specific health concerns.

The Role of SMN1 and SMN2 Genes in Spinal Muscular Atrophy

New Study: Zolgensma May Benefit Adults With SMA Types 2 and 3