Defining what matters: Patient and caregiver priorities for a core outcome set in type II GM1 gangliosidosis

Molecular Genetics and Metabolism 2026

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Authors: Zahra Nasseri Moghaddam, Maria Bleier, Sylvia Stöckler

DOI: 10.1016/j.ymgme.2025.109561

Cited by: 0

Fields of Study & Subjects

Lysosomal Storage Disorders Research Delphi Technique in Research Glycogen Storage Diseases and Myoclonus