Nitisinone (MDK-nitisinone) (MendeliKABS inc.) : indication : for the treatment of patients with hereditary tyrosinemia type 1 in combination with dietary restriction of tyrosine and phenylalanine

If untreated, survival in patients with HT-1 is less than 12 months of life; most of these children die as a result of liver failure and severe coagulopathy. The prevalence of HT-1 ranges from one in 12,000 to one in 100,000 individuals of Northern European descent. In Canada, higher prevalence (one...

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Bibliographic Details

Corporate Author:	Canadian Agency for Drugs and Technologies in Health
Format:	eBook
Language:	English
Published:	Ottawa (ON) Canadian Agency for Drugs and Technologies in Health 2018, April 2018
Series:	CADTH common drug review: clinical review report
Subjects:	Enzyme Inhibitors / Therapeutic Use Tyrosinemias / Drug Therapy Tyrosine / Toxicity Treatment Outcome Cyclohexanones / Therapeutic Use Nitrobenzoates / Therapeutic Use
Online Access:	https://www.ncbi.nlm.nih.gov/books/NBK533346
Collection:	National Center for Biotechnology Information - Collection details see MPG.ReNa