The SPINRAZA® (nusinersen) clinical trial program is the largest in spinal muscular atrophy (SMA) to date1*

Presymptomatic

NURTURE1

25 presymptomatic infants

Supportive, open-label trial

  • Phase 2 trial
  • 3 days to 42 days old at time of first dose
  • Duration: ~60 months
  • Primary endpoint: time to death or respiratory intervention

VIEW RESULTS

Infantile-onset

ENDEAR2,3

121 symptomatic infants
(Type 1)

Pivotal, controlled trial

  • Phase 3 trial; patients were randomized 2:1
  • ≤7 months of age at first dose (median 181 days)
  • Duration: 13 months
  • Primary endpoint: proportion of Hammersmith Infant Neurological Examination (HINE) Section 2 motor milestone responders

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Later-onset

CHERISH2,4

126 nonambulatory patients
(Types 2 and 3)

Pivotal, controlled trial

  • Phase 3 trial; patients were randomized 2:1
  • 2 to 9 years of age at screening
  • Duration: 15 months
  • Primary endpoint: change in Hammersmith Functional Motor Scale—Expanded (HFMSE)

VIEW RESULTS

*Trials have included 371 patients ranging in age from 3 days old to 16 years old at first dose.1

CS3A1

20 symptomatic infants
(Type 1)

Supportive, open-label trial

  • Phase 2 trial
  • ≥21 days to ≤7 months of age at screening
  • Duration: 45 months
  • Primary endpoint: proportion of HINE Section 2 motor milestone responders

CS2/CS121,5

28 ambulatory and nonambulatory patients (Types 2 and 3)

Supportive, open-label trial

  • 2 to 16 years of age at first dose
  • Duration: ~36 months
  • Primary endpoint: change in HFMSE

VIEW RESULTS

This is not a complete list of the SPINRAZA clinical trials.

Resources

Cameron
age 2.5
Infantile-onset (Type 1) SMA treated with SPINRAZA

Lauren
age 20
Later-onset (Type 3) SMA
treated with SPINRAZA

Cameron
age 2.5
Infantile-onset (Type 1) SMA
treated with SPINRAZA

Lauren
age 20
Later-onset (Type 3) SMA
treated with SPINRAZA

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