Work

Correlation of individual SMN protein with observed efficacy in the risdiplam SMA clinical trials

Year: 2024

Type: conference-paper

DOI: 10.1136/jnnp-2024-abn.184

Authors: Kletzl Heidemarie, Cleary Yumi, Grimsey Paul, Chiriboga Claudia A, Mercuri Eugenio, McIver Tammy, Thomas Riorge, Scalco Renata S

Institutions: Roche (Switzerland), Roche (United Kingdom), Columbia University Irving Medical Center

Language: English

Topic: Neurogenetic and Muscular Disorders Research

Subfield: Genetics

Field: Medicine

Domain: Health Sciences

SDG: Good health and well-being

Open Access status: closed

OpenAlex ID: W4404183311

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