Trial Profile
An Open-Label Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of Multiple Doses of ISIS 396443 Delivered Intrathecally to Subjects With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy
Status:
Active, no longer recruiting
Phase of Trial:
Phase II
Latest Information Update: 29 Sep 2023
Price :
$35
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At a glance
- Drugs Nusinersen (Primary)
- Indications Spinal muscular atrophy
- Focus Therapeutic Use
- Acronyms NURTURE
- Sponsors Biogen
- 01 Aug 2023 Results (data cut: February 15, 2021, additional 2 y of follow-up) assessing benefit of nusinersen initiated in the presymptomatic stage of spinal muscular atrophy published in the Muscle and Nerve
- 14 Mar 2022 Results published in the Biogen Media Release.
- 14 Mar 2022 According to a Biogen media release, latest results from the study will be presented at the 2022 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference.