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Clinical trial

A Global Study of a Single, One-Time Dose of AVXS-101 Delivered to Infants With Genetically Diagnosed and Pre-symptomatic Spinal Muscular Atrophy With Multiple Copies of SMN2

NCT03505099 is registered in ClinicalTrials.gov with Novartis Gene Therapies as lead sponsor.

NCT NCT03505099
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Registry record

As filed in ClinicalTrials.gov.

Title
A Global Study of a Single, One-Time Dose of AVXS-101 Delivered to Infants With Genetically Diagnosed and Pre-symptomatic Spinal Muscular Atrophy With Multiple Copies of SMN2
Lead sponsor
Novartis Gene Therapies
Phase
Phase 3
Overall status
Completed
Start date
April 2, 2018
Completion date
June 15, 2021
Enrollment
30 (actual)
Sites
16

Conditions

As the registry lists them; a condition with its own page is linked.

spinal muscular atrophy