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Efficacy and safety of once-daily nitisinone for patients with alkaptonuria (SONIA 2): an international, multicentre, open-label, randomised controlled trial

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作者单位: Liverpool Univ Hosp NHS Fdn Trust, Dept Clin Biochem & Metab Med, Liverpool L7 8XP, Merseyside [1] Univ Liverpool, Liverpool Clin Trials Ctr, Liverpool, Merseyside, England [2] Hop Necker Enfants Malad, Paris, France [3] Univ Siena, Dept Biotechnol Chem & Pharm, Siena, Italy [4] Liverpool Univ Hosp NHS Fdn Trust, Dept Ophthalmol, Liverpool, Merseyside, England [5] Swedish Orphan Biovitrum, Stockholm, Sweden [6] Liverpool Univ Hosp NHS Fdn Trust, Dept Physiotherapy, Liverpool, Merseyside, England [7] Univ Liverpool, Inst Ageing & Chron Dis, Liverpool, Merseyside, England [8] Liverpool Univ Hosp NHS Fdn Trust, Dept Cardiol, Liverpool, Merseyside, England [9] Liverpool Univ Hosp NHS Fdn Trust, Dept Clin Pharmacol, Liverpool, Merseyside, England [10] Nord Biosci, Herlev, Denmark [11] Slovak Acad Sci, Inst Clin & Translat Res, Biomed Res Ctr, Bratislava, Slovakia [12] Cudos, Hoofddorp, Netherlands [13] Liverpool John Moores Univ, Sch Sport & Exercise Sci, Liverpool, Merseyside, England [14] PSR Grp, Hoofddorp, Netherlands [15] Natl Inst Rheumat Dis, Piestany, Slovakia [16] Liverpool Univ Hosp NHS Fdn Trust, Dept Radiol, Liverpool, Merseyside, England [17] AKU Soc, Cambridge, England [18] Liverpool Univ Hosp NHS Fdn Trust, Dept Nucl Med, Liverpool, Merseyside, England [19] Liverpool Univ Hosp NHS Fdn Trust, Dept Ear Nose & Throat, Liverpool, Merseyside, England [20] Liverpool Univ Hosp NHS Fdn Trust, Dept Dermatol, Liverpool, Merseyside, England [21]
发布时间 2022-09-11
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