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GM1-gangliosidosis: The caregivers’ assessments of symptom impact and most important symptoms to treat
December 21, 2022
Article
Parental coping with uncertainties along the severe combined immunodeficiency journey
October 27, 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
Using a patient portal to increase enrollment in a newborn screening research study: Observational study
February 10, 2022