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Eftychia Eirini Psarelli on Muck Rack

Eftychia Eirini Psarelli

As seen in: The Lancet
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Articles

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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By Helen Bygott, Lakshminarayan R. Ranganath, Eftychia Eirini Psarelli, Jean-Baptiste Arnoux
| The Lancet Verified
Summary Background Alkaptonuria is a rare, genetic, multisystem disease characterised by the accumulation of homogentisic acid (HGA). No HGA-lowering therapy has been approved to date. The aim of SONIA 2 was to investigate the efficacy and safety of once-daily nitisinone for reducing HGA excretion in patients with alkaptonuria and to evaluate whether nitisinone has a clinical benefit.
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