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Journal Articles

in Metabolic Diseases
 101-99 of 99 Articles

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From The JAMA Network
JAMA
Original Investigation  | 
Effect of Oral Eliglustat on Splenomegaly in Patients With Gaucher Disease Type 1:  The ENGAGE Randomized Clinical Trial
Pramod K. Mistry, MD, PhD, FRCP; Elena Lukina, MD; Hadhami Ben Turkia, MD; et al.
JAMA Neurology
Original Investigation  | 
Deep Intronic GBE1 Mutation in Manifesting Heterozygous Patients With Adult Polyglucosan Body Disease
H. Orhan Akman, PhD; Or Kakhlon, PhD; Jorida Coku, MS; et al.
JAMA Surgery
Original Investigation  | 
Prediction of Nocturia Severity in Men:  Nocturnal Urine Overproduction vs Race or Metabolic Risk Factors
Daniel Nassau, MD; Svetlana Avulova, MD; Fara M. Friedman, MD; et al.
JAMA Dermatology
Observation  | 
Fanconi Syndrome Induced by Vemurafenib:  A New Renal Adverse Event
Daphné Denis, MD; Nathalie Franck, MD; Fanny Fichel, MD; et al.