What question did this study set out to answer?

To evaluate the safety and clinical efficacy of nusinersen in infants and children with infantile- or later-onset spinal muscular atrophy who were ineligible for the ENDEAR and CHERISH trials.

January 27, 2021Muscle & NerveOpen Access

Safety and efficacy of nusinersen in spinal muscular atrophy: The EMBRACE study

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To evaluate the safety and clinical efficacy of nusinersen in infants and children with infantile- or later-onset spinal muscular atrophy who were ineligible for the ENDEAR and CHERISH trials.
Participants were randomized 2:1 to intrathecal nusinersen (12-mg scaled equivalent dose; n = 14) or a sham control procedure (n = 7) during part 1 (~14 months; NCT02462759).
Participants subsequently received open-label intrathecal nusinersen for approximately 24 months during part 2 of the study.
Part 1 was terminated early due to demonstrated motor function benefits of nusinersen in the ENDEAR trial.
Motor milestone responder rates were 93% with nusinersen at the last available assessment, compared to 29% in the sham group during part 1 and 83% among those transitioned from sham to nusinersen in part 2.
No study discontinuations or adverse events were related to nusinersen; the most frequent adverse events were pyrexia, cough, pneumonia, and upper respiratory tract infections.

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Implication

Randomized trial shows higher motor milestone response rates and favorable safety with nusinersen in spinal muscular atrophy, indicating therapeutic benefit across diverse clinical presentations.

Cite This Study

Acsádi et al. (2021) studied this question.

synapsesocial.com/papers/6a10ef0e69716c70d0488ee1 https://doi.org/10.1002/mus.27187

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Implication

Randomized trial shows higher motor milestone response rates and favorable safety with nusinersen in spinal muscular atrophy, indicating therapeutic benefit across diverse clinical presentations.

Safety and efficacy of nusinersen in spinal muscular atrophy: The EMBRACE study

Key Points

Discussion

Implication

Cite This Study

Safety and efficacy of nusinersen in spinal muscular atrophy: The EMBRACE study

Key Points

Discussion

Implication

Cite This Study

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