Work

A phase 2 study assessing TAK-611150 mg intrathecal weekly in patients with late-infantile metachromatic leukodystrophy (SHP611–201; EMBOLDEN) compared to matched historical control data from children with late-infantile MLD (GLIA-MLD)

Year: 2024

Type: article

Source: Molecular Genetics and Metabolism

DOI: 10.1016/j.ymgme.2023.107744

Authors: Laura A. Adang, C.J. Malanga, Barbara K. Burton, Roberto Giugliani, Samuel Groeschel, Michal Inbar-Feigenberg, Caroline Sevin, Mireia Del Toro Riera, Shaoming Yin, Suchitrita Rathmann, David A.H. Whiteman, Adeline Vanderver

Institutions: Children's Hospital of Philadelphia, Northwestern University, Lurie Children's Hospital, Universidade Federal do Rio Grande do Sul, University Children's Hospital Tübingen, University of Tübingen, University of Toronto, Bicêtre Hospital, Vall d'Hebron Hospital Universitari

Language: English

Topic: Lysosomal Storage Disorders Research

Subfield: Physiology

Field: Medicine

Domain: Health Sciences

SDG: Good health and well-being

Open Access status: closed

OpenAlex ID: W4391378822

FWCI: 0.571

Cites:

Cited by: 2

Related to: 10