Trial Profile

An Expanded Access Program for Risdiplam in Patients With Type 1 or Type 2 Spinal Muscular Atrophy

Status: Completed

Phase of Trial: Clinical Phase Unknown

Latest Information Update: 05 May 2023

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At a glance

Drugs Risdiplam (Primary)
Indications Spinal muscular atrophy
Focus Expanded access; Therapeutic Use
Sponsors Genentech

Most Recent Events

01 May 2023 Results (n=6) assessing the use of risdiplam in a series of type 2 non-sitter patients aged 16 years and older with 5q spinal muscular atrophy, published in the Muscle and Nerve.
30 Sep 2020 Status changed from recruiting to completed.
13 Feb 2020 New trial record

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