News Nulibry Now Available for Molybdenum Cofactor Deficiency Type A By Brian Park, PharmD Publish Date April 19, 2021
News Sodium Bicarbonate May Mitigate Calcium Phosphate Stone Risk in CKD By Jody A. Charnow Publish Date April 19, 2021
Drugs in the Pipeline Tesomet Granted Orphan Drug Status for Prader-Willi Syndrome By Brian Park, PharmD Publish Date March 3, 2021
News Nulibry Approved for Molybdenum Cofactor Deficiency Type A By Brian Park, PharmD Publish Date March 1, 2021
WORLDSymposium 2021 Early Treatment With Galsulfase Appears to Slow Disease Progression in MPS VI By Cassandra Pardini, PharmD Publish Date February 11, 2021
WORLDSymposium 2021 Case Series Describes Hydroxychloroquine Use, Toxicity in Fabry Disease By Cassandra Pardini, PharmD Publish Date February 11, 2021
WORLDSymposium 2021 Clinical Benefits Observed With Investigational Gene Therapy for ES-EJ Metachromatic Leukodystrophy By Cassandra Pardini, PharmD Publish Date February 11, 2021
WORLDSymposium 2021 Does Gaucher Disease Up SARS-CoV-2 Infection Risk? By Diana Ernst, RPh Publish Date February 10, 2021
WORLDSymposium 2021 Positive Results for IV Hydroxypropyl Beta Cyclodextrin in Niemann-Pick Disease Type By Cassandra Pardini, PharmD Publish Date February 10, 2021
WORLDSymposium 2021 Enhanced Brain Connectivity Observed With Venglustat Plus Imiglucerase in GD3 By Brian Park, PharmD Publish Date February 10, 2021
WORLDSymposium 2021 Cardiac Efficacy Maintained With Long-Term Migalastat in Fabry Disease By Cassandra Pardini, PharmD Publish Date February 10, 2021
WORLDSymposium 2021 Olipudase Alfa Improves Lung Function, Spleen Volume in ASMD By Brian Park, PharmD Publish Date February 9, 2021
WORLDSymposium 2021 Patient With Gaucher Disease and ITP Effectively Treated With Eltrombopag By Diana Ernst, RPh Publish Date February 9, 2021
WORLDSymposium 2021 Arimoclomol Demonstrates Sustained Effect in Niemann-Pick Disease Type C By Lea Eslava-Kim, PharmD Publish Date February 9, 2021
WORLDSymposium 2021 Extending Enzyme Replacement Therapy Post-HCT in Hurler Syndrome Appears Beneficial By Cassandra Pardini, PharmD Publish Date February 9, 2021
WORLDSymposium 2021 Pegunigalsidase Alfa Improves Renal Function in Fabry Disease Switch-Over Trial By Brian Park, PharmD Publish Date February 9, 2021
WORLDSymposium 2021 Enzyme Replacement Therapy in Late-Onset Pompe Disease: Patient Outcomes Explored By Cassandra Pardini, PharmD Publish Date February 9, 2021