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The study was provided with ethical approval by the NHS Health Research Authority and local NHS Research Ethics Committee (Integrated Research Application System Project Code 186163, REC Reference 15\/EE\/0334). The study is registered on(Identifier; NCT02739217 -) and European Union Drug Regulating Authorities Clinical Trials (EudraCT Number 2015\u2013001625-16).Subjects are only included in this study if informed consent is obtained in advance of any study-specific procedures and documented in an IEC-approved information sheet and consent form. Given the nature and severity of Alstr\u00f6m Syndrome and the resultant loss of sight, subjects are not expected to be able to read a normal information sheet and consent form. 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The other authors declare no competing interest.","order":3,"name":"Ethics","group":{"name":"EthicsHeading","label":"Competing interests"}},{"value":"Springer Nature remains neutral with regard to jurisdictional claims in published maps and institutional affiliations.","order":4,"name":"Ethics","group":{"name":"EthicsHeading","label":"Publisher\u2019s Note"}}],"article-number":"88"}}