An Open-label, Escalating Dose Study to Assess the Safety, Tolerability and Dose-range Finding of a Single Intrathecal Dose of ISIS 396443 in Patients With Spinal Muscular Atrophy

Name: An Open-label, Escalating Dose Study to Assess the Safety, Tolerability and Dose-range Finding of a Single Intrathecal Dose of ISIS 396443 in Patients With Spinal Muscular Atrophy
Creator: Vivli
Published: 2025-06-01 01:03:34
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DataCite Commons2025-06-01 更新2026-05-07 收录

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https://search.vivli.org/doiLanding/studies/00002890/isLanding

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资源简介：

This objectives of this study are to evaluate the safety, tolerability, and pharmacokinetics of a single dose of nusinersen (ISIS 396443) administered intrathecally to participants with Spinal Muscular Atrophy (SMA).

提供机构：

Vivli

创建时间：

2018-10-27