Trusted Resources: Education
Scientific literature and patient education texts
Evidence of Treatment Benefits in Patients With Mucopolysaccharidosis Type I-Hurler in Long-Term Follow-Up Using a New Magnetic Resonance Imaging Scoring System
source: The Journal of pediatrics
year: 2022
authors: Pontesilli S,Baldoli C,Rosa PAD,Cattoni A,Bernardo ME,Meregalli P,Gasperini S,Motta S,Fumagalli F,Tucci F,Baciga F,Consiglieri G,Canonico F,De Lorenzo P,Chiapparini L,Gentner B,Aiuti A,Biondi A,Rovelli A,Parini R
summary/abstract:We developed a brain and spine magnetic resonance scoring system based on a magnetic resonance assessment of 9 patients with mucopolysaccharidosis type I-Hurler who underwent hematopoietic stem-cell transplantation. The score is reliable and correlates with long-term clinical and cognitive outcome in patients with mucopolysaccharidosis type I-Hurler.
organization: Department of Neuroradiology, IRCCS San Raffaele Scientific Institute, Milano, Italy.DOI: 10.1016/j.jpeds.2021.09.020
read more
BookmarkRelated Content
-
Stewart RustDr Rust completed his first degree at Le...
-
Gene Therapy May Reverse Hurler Syndrome, a Rare and Severe Illness in KidsGene therapy might soon offer a new opti...
-
University of Minnesota – Mucopolysaccharidosis (MPS) CenterOur MPS Center is internationally recogn...
-
UCSF – Lysosomal Storage Disease CenterThe UCSF Lysosomal Storage Disease Cente...
-
Hematopoietic Stem- and Progenitor-Cell Gene Therapy for Hurler SyndromeAllogeneic hematopoietic stem-cell trans...
-
Mucopolysaccharide Storage Disease Type I: Hurler, Hurler-Scheie, and Scheie Syndromeshttps://www.youtube.com/watch?v=en9M2P8S...
-
A Guide to Understanding MPS IMPS I is a mucopolysaccharide disease al...
Powered by
