Inotersen for treating hereditary transthyretin amyloidosis (HST9)
Evidence-based recommendations on inotersen (Tegsedi) for stage 1 and stage 2 polyneuropathy in adults with hereditary transthyretin amyloidosis
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Burosumab for treating X-linked hypophosphataemia in children and young people (HST8)
Evidence-based recommendations on burosumab (Crysvita) for treating X-linked hypophosphataemia (XLH) in children and young people
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Strimvelis for treating adenosine deaminase deficiency–severe combined immunodeficiency (HST7)
Evidence-based recommendation on Strimvelis for adenosine deaminase deficiency–severe combined immunodeficiency
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Asfotase alfa for treating paediatric-onset hypophosphatasia (HST6)
Evidence-based recommendations on asfotase alfa (Strensiq) for treating paediatric-onset hypophosphatasia in adults and children
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Eliglustat for treating type 1 Gaucher disease (HST5)
Evidence-based recommendations on eliglustat (Cerdelga) for treating type 1 Gaucher disease in adults
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Migalastat for treating Fabry disease (HST4)
Evidence-based recommendations on migalastat (Galafold) for treating Fabry disease in people over 16