Validation of the shortened Hunter Syndrome-Functional Outcomes for Clinical Understanding Scale (HS-FOCUS)

by Maria Mattera, Margaret K. Vernon, Mireia Raluy-Callado, Jaromir Mikl
Published 2018-11-01

Assessing the impact on caregivers caring for patients with rare pediatric lysosomal storage diseases: development of the Caregiver Impact Questionnaire

by Magdalena Harrington, Asha Hareendran, Anne Skalicky, Hilary Wilson, Marci Clark, Jaromir Mikl
Published 2019-07-01