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A father's fight to help his sons - and fix clinical trials.
- Source :
-
Nature [Nature] 2019 Jan; Vol. 565 (7738), pp. 148-151. - Publication Year :
- 2019
- Subjects :
- Adolescent
Alkaptonuria genetics
Alkaptonuria metabolism
Alkaptonuria urine
Animals
Autopsy
Child
Clinical Trials as Topic economics
Cyclohexanones pharmacology
Cyclohexanones therapeutic use
Disease Models, Animal
Disease Progression
Drug Approval
Endpoint Determination
Female
Homogentisic Acid metabolism
Humans
Infant
Male
Mice
National Health Programs
National Institutes of Health (U.S.)
Nitrobenzoates pharmacology
Nitrobenzoates therapeutic use
Observational Studies as Topic
Off-Label Use
Orphan Drug Production
Rare Diseases genetics
Rare Diseases metabolism
Rare Diseases urine
Rats
Sample Size
Tyrosine metabolism
Tyrosinemias drug therapy
Tyrosinemias enzymology
United States
United States Food and Drug Administration legislation & jurisprudence
Alkaptonuria drug therapy
Clinical Trials as Topic methods
Clinical Trials as Topic standards
Compassionate Use Trials
Rare Diseases drug therapy
Subjects
Details
- Language :
- English
- ISSN :
- 1476-4687
- Volume :
- 565
- Issue :
- 7738
- Database :
- MEDLINE
- Journal :
- Nature
- Publication Type :
- Academic Journal
- Accession number :
- 30626961
- Full Text :
- https://doi.org/10.1038/d41586-019-00035-x