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Article
GM1-gangliosidosis: The caregivers’ assessments of symptom impact and most important symptoms to treat
December 21, 2022
Article
Using a patient portal to increase enrollment in a newborn screening research study: Observational study
February 10, 2022
Article
Caregivers' assessment of meaningful and relevant clinical outcome assessments for Sanfilippo syndrome
April 25, 2022
Article
Preparing newborn screening for the future: A collaborative stakeholder engagement exploring challenges and opportunities to modernizing the newborn screening system
February 12, 2022
Article
Parental coping with uncertainties along the severe combined immunodeficiency journey
October 27, 2022