Published guidance, NICE advice and quality standards

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Published guidance, NICE advice and quality standards
Title	Reference number	Published	Last updated
Voretigene neparvovec for treating inherited retinal dystrophies caused by RPE65 gene mutations	HST11	9 October 2019	9 October 2019
Patisiran for treating hereditary transthyretin amyloidosis	HST10	14 August 2019	14 August 2019
Inotersen for treating hereditary transthyretin amyloidosis	HST9	22 May 2019	22 May 2019
Burosumab for treating X-linked hypophosphataemia in children and young people	HST8	10 October 2018	10 October 2018
Strimvelis for treating adenosine deaminase deficiency–severe combined immunodeficiency	HST7	7 February 2018	7 February 2018
Eliglustat for treating type 1 Gaucher disease	HST5	28 June 2017	28 June 2017
Migalastat for treating Fabry disease	HST4	22 February 2017	22 February 2017
Eculizumab for treating atypical haemolytic uraemic syndrome	HST1	28 January 2015	28 January 2015