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Patient-reported outcomes and functional assessments of patients with Alkaptonuria in a 3-year Nitisinone treatment trial.

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Title: Patient-reported outcomes and functional assessments of patients with Alkaptonuria in a 3-year Nitisinone treatment trial.
Authors: Spears KR; Human Biochemical Genetics Section, Medical Genetics Branch, NHGRI, United States., Rossignol F; Human Biochemical Genetics Section, Medical Genetics Branch, NHGRI, United States., Perry MB; Rehabilitation Medicine Department, NIH Clinical Center, United States., Kayser MA; Human Biochemical Genetics Section, Medical Genetics Branch, NHGRI, United States., Suwannarat P; Human Biochemical Genetics Section, Medical Genetics Branch, NHGRI, United States., O'Brien KE; Office of the Clinical Director, NHGRI, United States., Bryant JC; Office of the Clinical Director, NHGRI, United States., Greenwood WF; Cycle Pharmaceuticals Limited, United Kingdom., Fuller S; Cycle Pharmaceuticals Limited, United Kingdom., Gahl WA; Human Biochemical Genetics Section, Medical Genetics Branch, NHGRI, United States., Introne WJ; Human Biochemical Genetics Section, Medical Genetics Branch, NHGRI, United States. Electronic address: wintrone@mail.nih.gov.
Source: Molecular genetics and metabolism [Mol Genet Metab] 2024 Sep-Oct; Vol. 143 (1-2), pp. 108562. Date of Electronic Publication: 2024 Aug 03.
Publication Type: Journal Article; Randomized Controlled Trial
Journal Info: Publisher: Academic Press Country of Publication: United States NLM ID: 9805456 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1096-7206 (Electronic) Linking ISSN: 10967192 NLM ISO Abbreviation: Mol Genet Metab Subsets: MEDLINE
Database: MEDLINE Ultimate
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  Data: Patient-reported outcomes and functional assessments of patients with Alkaptonuria in a 3-year Nitisinone treatment trial.
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  Data: <searchLink fieldCode="AU" term="%22Spears+KR%22">Spears KR</searchLink>; Human Biochemical Genetics Section, Medical Genetics Branch, NHGRI, United States.<br /><searchLink fieldCode="AU" term="%22Rossignol+F%22">Rossignol F</searchLink>; Human Biochemical Genetics Section, Medical Genetics Branch, NHGRI, United States.<br /><searchLink fieldCode="AU" term="%22Perry+MB%22">Perry MB</searchLink>; Rehabilitation Medicine Department, NIH Clinical Center, United States.<br /><searchLink fieldCode="AU" term="%22Kayser+MA%22">Kayser MA</searchLink>; Human Biochemical Genetics Section, Medical Genetics Branch, NHGRI, United States.<br /><searchLink fieldCode="AU" term="%22Suwannarat+P%22">Suwannarat P</searchLink>; Human Biochemical Genetics Section, Medical Genetics Branch, NHGRI, United States.<br /><searchLink fieldCode="AU" term="%22O'Brien+KE%22">O'Brien KE</searchLink>; Office of the Clinical Director, NHGRI, United States.<br /><searchLink fieldCode="AU" term="%22Bryant+JC%22">Bryant JC</searchLink>; Office of the Clinical Director, NHGRI, United States.<br /><searchLink fieldCode="AU" term="%22Greenwood+WF%22">Greenwood WF</searchLink>; Cycle Pharmaceuticals Limited, United Kingdom.<br /><searchLink fieldCode="AU" term="%22Fuller+S%22">Fuller S</searchLink>; Cycle Pharmaceuticals Limited, United Kingdom.<br /><searchLink fieldCode="AU" term="%22Gahl+WA%22">Gahl WA</searchLink>; Human Biochemical Genetics Section, Medical Genetics Branch, NHGRI, United States.<br /><searchLink fieldCode="AU" term="%22Introne+WJ%22">Introne WJ</searchLink>; Human Biochemical Genetics Section, Medical Genetics Branch, NHGRI, United States. Electronic address: wintrone@mail.nih.gov.
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      – TitleFull: Patient-reported outcomes and functional assessments of patients with Alkaptonuria in a 3-year Nitisinone treatment trial.
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              Text: 2024 Sep-Oct
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