Trials for later-onset SMA

CHERISH was a phase 3, multicenter, randomized (2:1), double-blind, sham procedure–controlled study1,2 :

  • 126 patients with genetically confirmed spinal muscular atrophy (SMA) aged 2 to 9 years at screening (symptom onset after 6 months of age), with the ability to sit independently, and with no history of independent walking1
  • Based on the positive results of the interim analysis, the trial was ended early and all patients were enrolled in SHINE, the open-label extension trial1


Patients were administered treatment on days 1, 29, and 85, followed by a maintenance dose on day 274. This administration differs from the approved SPINRAZA® (nusinersen) dosing regimen.1,2

Clinically meaningful improvements in motor function1

Improved upper limb function1

  • The most common adverse reactions that occurred in later-onset trials were pyrexia, headache, vomiting, and back pain2

 

Image of Ruby aged 6 with Type 2 SMA painting

Individual results may vary based on several factors, including severity of disease, initiation of treatment, and duration of therapy.

Ruby
age 4

Later-onset (Type 2) SMA
treated with SPINRAZA

Ruby
age 4

Later-onset (Type 2) SMA
treated with SPINRAZA

Individual results may vary based on several factors, including severity of disease, initiation of treatment, and duration of therapy.