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Top Line Results of the Phase 1b, Open-label, Multiple Ascending Dose Proof of Concept Study of the PIKfyve Inhibitor VRG50635 in Familial and Sporadic Amyotrophic Lateral Sclerosis (P11-7.013)

Year: 2026

Type: article

Source: Neurology

DOI: 10.1212/wnl.0000000000216449

Authors: Diego Cadavid, Robert Scannevin, Ines Hoffmann, Anil Tarachandani, Irene Choi, Ashwin Swami, Joanna Haas, Martin Schmidt, Brian Shook, Madelyn Rubenstein, Shane Raines, Philip Van Damme, Pentti Tienari, Eino Solje, Manu Jokela, Angela Genge, Colleen O’Connell, Ruben van Eijk, Leonard Van den Berg

Institutions: Verge Genomics (United States), Universitair Ziekenhuis Leuven, Helsinki University Hospital, University of Eastern Finland, Turku University Hospital, Montreal Neurological Institute and Hospital, University Medical Center Utrecht

Language: English

Topic: Amyotrophic Lateral Sclerosis Research

Subfield: Neurology

Field: Medicine

Domain: Health Sciences

SDG: Good health and well-being

Open Access status: closed

OpenAlex ID: W7164186217

FWCI:

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