Spinal Muscular Atrophy

Updates in Treatment of Adult Spinal Muscular Atrophy

Challenges and Considerations in Treatment of Adult Spinal Muscular Atrophy

Key opinion leader discusses considerations for adult patients with spinal muscular atrophy (SMA) including disease burden, treatment evaluation, and the use of real-world evidence.

Phase 2 Trial Drug Apitegromab Shows Progress for Spinal Muscular Atrophy Patients

March 17, 2021

The phase 2 TOPAZ trial demonstrated treatment is safe and improves motor function in patients with SMA.

Spinal Muscular Atrophy Drug Risdiplam Proves Success in Infants

March 02, 2021

Patients treated with risdiplam at 12 months demonstrated significant improvement in survival and developmental milestones, with 19 of 21 (90%) infants able to survive without permanent ventilation and 7 (41%) infants able to sit without support for at least 5 seconds.

FDA Approves Evrysdi, Third Drug for Spinal Muscular Atrophy

August 10, 2020

FDA Updates: Assays Used in Cancer Care Get Green Light

August 09, 2020

Adaptive Biotechnologies’ clonoSEQ gets third approval; FDA also acts on first at-home treatment for spinal muscular atrophy.