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City Danbury / Franklin / / Company Rare Disease Therapeutics Inc. / / Country Norway / United States / Canada / / / Event Product Issues / / Facility Pathology Laval University / / / IndustryTerm Rare Disease Therapeutics / food / energy / chemical processes / / MedicalCondition recessive disorder / untreated tyrosinemia type / hypertrophic cardiomyopathy / rare genetic metabolic disorder / rare diseases / past / tyrosinemia type / Renal Fanconi syndrome / Urea Cycle Disorders / Acute polyneuropathy / liver failure / pains in the legs / cirrhosis / potential liver cancer / Progressive cirrhosis / tyrosinemia / rare disorder / disorder / chronic liver failure / tyrosinemia type I / rickets / diarrhea / severe liver disease / Metabolic disorders / chronic form / Primary Lateral Sclerosis / Pompe Disease / end stage liver failure / hepatorenal tyrosinemia / acute life-threatening liver failure / tyrosinemia type / Hereditary Ataxia / hypertension / disease / kidney dysfunction / Ornithine Transcarbamylase Deficiency / hepatocellular carcinoma / Fever Diarrhea/bloody / recessive disorders / experience treating tyrosinemia type / Giant Hypertrophic Gastritis / Amyloidosis / untreated / tyrosinemia type / Multiple System Atrophy / pain / vomiting / Medullary Thyroid Cancer / liver cancer / rare disorders / frequent nosebleeds / Menetrier’s Disease / disorders / heart muscle weakness / dehydration / / MedicalTreatment Liver transplant / counseling / low protein diet / low-protein diet / / Organization Laval University / National Organization for Rare Disorders NORD Guides for Physicians / Department of Molecular Biology / U.S. Food and Drug Administration / National Organization for Rare Disorders / / Person ROBERT M. TANGUAY / Danielle Barckett / / / Position Physician / Professor / Pediatrician / following physician / / Product Orfadin / gene / / ProvinceOrState Quebec / Connecticut / Tennessee / / PublishedMedium Molecular Biology / / Technology transplantation / / URL www.rarediseases.org / www.raretx.com / / SocialTag