Consensus guidelines for the monitoring and management of metachromatic leukodystrophy in the United States
Laura Adang, Joshua L. Bonkowsky, Jaap Jan Boelens, Eric J. Mallack, Rebecca C. Ahrens‐Nicklas, John Bernat, Annette Bley, Barbara K. Burton, Alejandra Darling, Florian Eichler, Erik A. Eklund, Lisa Emrick, Maria L. Escolar, Ali Fatemi, Jamie L. Fraser, Amy Gaviglio, Stephanie Keller, Marc C. Patterson, Paul J. Orchard, Jennifer Orthmann‐Murphy, Jonathan D. Santoro, Lüdger Schöls, Caroline Sevin, Isha N. Srivastava, Deepa Rajan, Jennifer Rubin, Keith Van Haren, Melissa Wasserstein, Ayelet Zerem, Francesca Fumagalli, Lucia Laugwitz, Adeline Vanderver
Topics & Concepts
Metachromatic leukodystrophyMedicineDiseaseArylsulfatase AIntensive care medicineLeukodystrophyGenetic testingClinical trialNewborn screeningPediatricsPathologyInternal medicineLysosomal Storage Disorders ResearchMetabolism and Genetic DisordersBiochemical and Molecular Research