Trial for infantile-onset
spinal muscular atrophy (SMA)

ENDEAR

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

  • 121 genetically confirmed symptomatic infants with symptom onset before 6 months of age and who were 7 months of age or younger at first dose were included1
  • 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 trial3
  • Not all patients completed the day 394 assessment. Efficacy data were calculated at the later of days 183, 302, or 3944


Patients in the SPINRAZA arm had earlier onset of SMA, more respiratory issues, and more difficulties swallowing or feeding at baseline.

Event-free survival1

Reduced risk of
permanent ventilation2

Improved motor function
and motor milestones2

  • The most common adverse reactions that occurred in ENDEAR were lower respiratory infection and constipation. Serious adverse reactions of atelectasis were more frequent in SPINRAZA-treated patients (18%) than in control patients (10%)1

 

Image of Sofia aged 2.5 playing in a pile of leaves

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

Cameron
age 2.5

Infantile-onset (Type 1) SMA
treated with SPINRAZA

Cameron
age 2.5

Infantile-onset (Type 1) SMA
treated with SPINRAZA

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