Trial Profile

A Phase II, Open-label Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy

Status: Recruiting

Phase of Trial: Phase II

Latest Information Update: 20 Feb 2026

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At a glance

Drugs Risdiplam (Primary)
Indications Spinal muscular atrophy
Focus Adverse reactions; Pharmacokinetics
Acronyms PUPFISH
Sponsors Roche

Most Recent Events

09 Jan 2026 Planned End Date changed from 17 Apr 2026 to 30 Nov 2026.
09 Jan 2026 Planned primary completion date changed from 17 Apr 2026 to 30 Nov 2026.
16 Sep 2025 Planned End Date changed from 30 Oct 2025 to 17 Apr 2026.

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