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NCT06019637RECRUITING

A Long-term Safety Study in Brazilian Patients With a Diagnosis of Spinal Muscular Atrophy Treated With Zolgensma

Study Summary

A long-term safety study in Brazilian patients with a confirmed diagnosis of Spinal Muscular Atrophy (SMA) treated with Onasemnogene Abeparvovec (Zolgensma®)

Conditions
Spinal Muscular Atrophies
Interventions / Drugs
Onasemnogene Abeparvovec
View on ClinicalTrials.gov ↗
Sponsor
NVS
Novartis AG
Diversified Pharma
D36/100
Study Details
Enrollment50 participants
Primary Completion—
Lead SponsorNovartis Pharmaceuticals
Data Retrieved2026-04-11 02:55:49.151661+00:00
What This Trial Means for Biotech Investors
Trial Phase Context
Status & Expected Data

Currently recruiting: the trial is enrolling patients. A data readout is not expected until after enrollment closes and the follow-up period is complete.

Enrolled: 50 participants
Sponsor Catalyst Profile

Novartis AG (NVS) is the sponsoring company for this trial. BiotechSign currently grades this company D (36/100) based on composite catalyst signals across its full pipeline. This trial is one data point in that overall catalyst picture.

NVS Full Pipeline →All Companies →
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