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Pediatrics Physician in Pittsburgh, PA
NPI: 1366552812Primary Employer
University of Pittsburgh Physicians
chp.edu
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Get Started for Freearrow_outwardNC State Medical License
Newborn screening for Krabbe disease in New York State: the first eight years' experience.
Authors: Joanne Kurtzberg, Natasha Shur, Patricia Galvin-Parton, Melissa Wasserstein, David Kronn, Georgianne Arnold, Darius Adams, Jennifer Kwon, Alejandro Iglesias, Joan Pellegrino
Journal: Genet Med
Publication Date: 2016-01-21
Early treatment is associated with improved cognition in Hurler syndrome.
Authors: Poe, Michele D, Chagnon, Sarah L, Escolar, Maria L
Journal: Ann Neurol
Publication Date: 2014-10-08
Early disease progression of Hurler syndrome.
Lead Sponsor: Ultragenyx Pharmaceutical Inc
Collaborators: Abeona Therapeutics, Inc
Intervention / Treatment: BIOLOGICAL: UX111, DRUG: Prophylactic Immunomodulatory (IM) Therapy, DRUG: Optimized Prophylactic IM Therapy, DRUG: Adjuvant IM Therapy
Lead Sponsor: Denali Therapeutics Inc.
Intervention / Treatment: DRUG: tividenofusp alfa
Lead Sponsor: University of Pittsburgh
Intervention / Treatment: OTHER: Palliative Care, BIOLOGICAL: Hematopoetic Stem Cell Transplantation