Long-Term Results from a Trial in Presymptomatic SMA Patients

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Updates from The Long-Term Results from a Trial in Presymptomatic SMA Patients

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Review the latest long-term results from a trial that focuses on a treatment option for presymptomatic infants with spinal muscular atrophy.

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Important Safety Information below and full Prescribing Information.

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  • Overview

    Updates from the NURTURE trial show long-term results with up to 5.7 years of follow-up in patients with spinal muscular atrophy (SMA) treated with SPINRAZA (nusinersen). To dive deeper into this topic, Dr. Jennifer Caudle is joined by pediatric neurologists Drs. Thomas Crawford and Britton Zuccarelli. Together, they discuss the results of this trial and considerations for treating infants with pre-symptomatic SMA. 


    SPINRAZA® (nusinersen) 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 safety information.

    © 2023 Biogen. All rights reserved. SPZ-US-5258 02/23

Schedule21 Mar 2023