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Gaucher disease type 3 from infancy through adulthood: a conceptual model of signs, symptoms, and impacts associated with ataxia and cognitive impairment by Raphael Schiffmann, James Turnbull, Robert Krupnick, Ruth Pulikottil-Jacob, Chad Gwaltney, Alaa Hamed, Isabela Batsu, Walter Heine, Eugen Mengel
Published 2025-04-01Get full text
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Initial Psychometric Evaluation of the Barth Syndrome Symptom Assessment (BTHS-SA) for Adolescents and Adults in a Phase 2 Clinical Study by Chad Gwaltney, Alan Shields, Emily Love, Sarah Ollis, Jonathan Stokes, Iyar Mazar, Ethan Arenson, Anthony Aiudi, R. J. Wirth, Carrie Houts
Published 2025-04-01Get full text
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