Infantile-onset (Type 1)
Controlled phase 3 trials ENDEAR:
(N=121, 13 months)1-3
  • Primary endpoint: event-free survival, proportion of patients meeting the criteria for motor milestone responder using HINE Section 2
  • Secondary endpoints: Overall survival, CHOP INTEND, CMAP, percentage of infants not requiring permanent ventilation, event-free survival in patients with disease duration of ≤12 weeks and >12 weeks
  • Additional assessment: safety
Patient characteristics
Supportive, phase 2, open-label trials CS3A:
(N=20, 45 months)2
  • Primary endpoint: proportion of patients achieving a level of improvement in motor milestones according to HINE Section 2*
  • Other endpoints: CHOP INTEND, event-free survival, safety and tolerability
Later-onset (Type 2 and Type 3)
Controlled phase 3 trials CHERISH:
(N=126, 15 months)4
  • Primary endpoint: change from baseline in HFMSE score at month 15
  • Secondary endpoints: HFMSE (≥3-point change), WHO motor milestones, RULM, standing alone, walking with assistance
  • Additional assessment: safety
Baseline characteristics
Supportive, phase 2, open-label trials CS2/CS12: longitudinal analysis across 2 studies
(N=28, 35 months)2
  • Primary endpoint: safety and tolerability of SPINRAZA administered intrathecally
  • Other endpoints: HFMSE, ULM, 6MWT
Presymptomatic (genetically diagnosed)
Controlled phase 3 trials Supportive, phase 2, open-label trials NURTURE/CS5:
(N=17, 28 months)2
  • Primary endpoint: time to death or respiratory ventilation
  • Other endpoints: HINE Section 2, CHOP INTEND, WHO motor milestones, safety and tolerability

HINE=Hammersmith Infant Neurological Examination; CHOP INTEND=Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders; CMAP=compound muscle action potential; HFMSE=Hammersmith Functional Motor Scale—Expanded; WHO=World Health Organization; RULM=Revised Upper Limb Module; ULM=Upper Limb Module; 6MWT=6-Minute Walk Test.

*Endpoint included voluntary grasp.
This is not a complete list of SPINRAZA clinical trials.



Key Icon

The SPINRAZA clinical trial program is the largest in spinal muscular atrophy to date.2†

Image of Cameron aged 2.5 years old with Type 1 SMA Image of Cameron aged 2.5 years old with Type 1 SMA Image of Ruby aged 4 years old with Type 2 SMA Image of Lauren aged 20 years old with Type 3 SMA
age 2.5

Infantile-onset (Type 1) SMA
treated with SPINRAZA

age 4

Later-onset (Type 2) SMA
treated with SPINRAZA

age 20

Later-onset (Type 3) SMA
treated with SPINRAZA

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


SPINRAZA is indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients.


Coagulation abnormalities and thrombocytopenia, including acute severe thrombocytopenia, have been observed after administration of some antisense oligonucleotides. Patients may be at increased risk of bleeding complications.

In the sham-controlled studies for patients with infantile-onset and later-onset SMA, 24 of 146 SPINRAZA-treated patients (16%) with high, normal, or unknown platelet count at baseline developed a platelet level below the lower limit of normal, compared to 10 of 72 sham-controlled patients (14%). Two SPINRAZA-treated patients developed platelet counts <50,000 cells per microliter, with the lowest level of 10,000 cells per microliter recorded on study day 28.

Renal toxicity, including potentially fatal glomerulonephritis, has been observed after administration of some antisense oligonucleotides. SPINRAZA is present in and excreted by the kidney. In the sham-controlled studies for patients with infantile-onset and later-onset SMA, 71 of 123 SPINRAZA-treated patients (58%) had elevated urine protein, compared to 22 of 65 sham-controlled patients (34%).

Laboratory testing and monitoring to assess safety should be conducted. Perform a platelet count, coagulation laboratory testing, and quantitative spot urine protein testing at baseline and prior to each dose of SPINRAZA and as clinically needed.

Severe hyponatremia was reported in an infant treated with SPINRAZA requiring salt supplementation for 14 months.

Cases of rash were reported in patients treated with SPINRAZA.

SPINRAZA may cause a reduction in growth as measured by height when administered to infants, as suggested by observations from the controlled study. It is unknown whether any effect of SPINRAZA on growth would be reversible with cessation of treatment.

The most common adverse reactions (≥20% of SPINRAZA-treated patients and ≥5% more frequently than in control patients) that occurred in the infantile-onset controlled study were lower respiratory infection and constipation. Serious adverse reactions of atelectasis were more frequent in SPINRAZA-treated patients (18%) than in control patients (10%). Because patients in this controlled study were infants, adverse reactions that are verbally reported could not be assessed. The most common adverse reactions that occurred in the later-onset controlled study were pyrexia, headache, vomiting, and back pain. Post-lumbar puncture syndrome has also been observed after the administration of SPINRAZA.

Please see full Prescribing Information for additional Important Safety Information.

As a courtesy, our full Prescribing Information is also available en Español. For prescribing decisions, please refer to official approved labeling.