Nitisinone
- Generic Name
- Nitisinone
- Brand Names
- Nityr, Orfadin
- Drug Type
- Small Molecule
- Chemical Formula
- C14H10F3NO5
- CAS Number
- 104206-65-7
- Unique Ingredient Identifier
- K5BN214699
- Background
Nitisinone is a synthetic reversible inhibitor of 4-hydroxyphenylpyruvate dioxygenase. It is used in the treatment of hereditary tyrosinemia type 1. It is sold under the brand name Orfadin.
- Indication
Used as an adjunct to dietary restriction of tyrosine and phenylalanine in the treatment of hereditary tyrosinemia type 1.
- Associated Conditions
- Tyrosinemia Type 1
- Associated Therapies
- -
Taste and Palatability of Orfadin Suspension
- First Posted Date
- 2012-11-28
- Last Posted Date
- 2014-11-06
- Lead Sponsor
- Swedish Orphan Biovitrum
- Target Recruit Count
- 18
- Registration Number
- NCT01734889
- Locations
- 🇩🇪
Klinikum der Universität München, München, Germany
🇫🇷Hopital Necker, Paris, France
🇩🇪Universitätsklinikum Düsseldorf, Düsseldorf, Germany
Nitisinone (NTBC) In Different Age Groups Of Patients With Alkaptonuria
- First Posted Date
- 2011-07-08
- Last Posted Date
- 2021-04-22
- Lead Sponsor
- University of California, San Diego
- Target Recruit Count
- 8
- Registration Number
- NCT01390077
- Locations
- 🇺🇸
University of California San Diego, La Jolla, California, United States
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