Page last updated: 2024-09-03

nitisinone and Orphan Diseases

nitisinone has been researched along with Orphan Diseases in 2 studies

Research

Studies (2)

Timeframe	Studies, this research(%)	All Research%
pre-1990	0 (0.00)	18.7374
1990's	0 (0.00)	18.2507
2000's	0 (0.00)	29.6817
2010's	2 (100.00)	24.3611
2020's	0 (0.00)	2.80

Authors

Authors	Studies
Adam, D	1
Aldamiz-Echevarria, LJ; Bliksrud, YT; Brunner-Krainz, M; Cocho de Juan, JA; Couce Pico, ML; Das, AM; de Baulny, HO; de Laet, C; Dionisi-Vici, C; Endig, J; Ernst, S; Freisinger, P; Gautschi, M; Gokcay, G; Hochuli, M; Jordan, J; Lotz-Havla, AS; Maiorana, A; Mandel, H; Mayorandan, S; McKiernan, P; Meyer, U; Moeslinger, D; Morlot, S; Sander, J; Santer, R; Scholl-Bürgi, S; Segarra, NG; Spiekerkoetter, U; Thimm, E; van Spronsen, F; Vogel, A; Zeman, J	1

Studies

Adam, D

Aldamiz-Echevarria, LJ; Bliksrud, YT; Brunner-Krainz, M; Cocho de Juan, JA; Couce Pico, ML; Das, AM; de Baulny, HO; de Laet, C; Dionisi-Vici, C; Endig, J; Ernst, S; Freisinger, P; Gautschi, M; Gokcay, G; Hochuli, M; Jordan, J; Lotz-Havla, AS; Maiorana, A; Mandel, H; Mayorandan, S; McKiernan, P; Meyer, U; Moeslinger, D; Morlot, S; Sander, J; Santer, R; Scholl-Bürgi, S; Segarra, NG; Spiekerkoetter, U; Thimm, E; van Spronsen, F; Vogel, A; Zeman, J

Other Studies

2 other study(ies) available for nitisinone and Orphan Diseases

Article	Year
A father's fight to help his sons - and fix clinical trials. Nature, 2019, Volume: 565, Issue:7738 Topics: Adolescent; Alkaptonuria; Animals; Autopsy; Child; Clinical Trials as Topic; Compassionate Use Trials; Cyclohexanones; Disease Models, Animal; Disease Progression; Drug Approval; Endpoint Determination; Female; Homogentisic Acid; Humans; Infant; Male; Mice; National Health Programs; National Institutes of Health (U.S.); Nitrobenzoates; Observational Studies as Topic; Off-Label Use; Orphan Drug Production; Rare Diseases; Rats; Sample Size; Tyrosine; Tyrosinemias; United States; United States Food and Drug Administration	2019
Cross-sectional study of 168 patients with hepatorenal tyrosinaemia and implications for clinical practice. Orphanet journal of rare diseases, 2014, Aug-01, Volume: 9 Topics: Adolescent; Child; Child, Preschool; Cross-Sectional Studies; Cyclohexanones; Enzyme Inhibitors; Female; Follow-Up Studies; Humans; Infant; Infant, Newborn; Liver Failure; Liver Transplantation; Male; Neonatal Screening; Nitrobenzoates; Rare Diseases; Renal Insufficiency; Retrospective Studies; Surveys and Questionnaires; Treatment Outcome; Tyrosinemias; Young Adult	2014

Article

Year

A father's fight to help his sons - and fix clinical trials.

Nature, 2019, Volume: 565, Issue:7738

Topics: Adolescent; Alkaptonuria; Animals; Autopsy; Child; Clinical Trials as Topic; Compassionate Use Trials; Cyclohexanones; Disease Models, Animal; Disease Progression; Drug Approval; Endpoint Determination; Female; Homogentisic Acid; Humans; Infant; Male; Mice; National Health Programs; National Institutes of Health (U.S.); Nitrobenzoates; Observational Studies as Topic; Off-Label Use; Orphan Drug Production; Rare Diseases; Rats; Sample Size; Tyrosine; Tyrosinemias; United States; United States Food and Drug Administration

2019

Cross-sectional study of 168 patients with hepatorenal tyrosinaemia and implications for clinical practice.

Orphanet journal of rare diseases, 2014, Aug-01, Volume: 9

Topics: Adolescent; Child; Child, Preschool; Cross-Sectional Studies; Cyclohexanones; Enzyme Inhibitors; Female; Follow-Up Studies; Humans; Infant; Infant, Newborn; Liver Failure; Liver Transplantation; Male; Neonatal Screening; Nitrobenzoates; Rare Diseases; Renal Insufficiency; Retrospective Studies; Surveys and Questionnaires; Treatment Outcome; Tyrosinemias; Young Adult

2014