Patient and caregiver experiences of living with acute hepatic porphyria in the UK: a mixed-methods study

Orphanet Journal of Rare Diseases

Stephen Lombardelli¹  Madeline Merkel²  Jordanna Mora²  John J. Ko²  Stephen Meninger²  Nicola Mason³  Marieke Schurer⁴  John Chamberlayne⁵  Sue Burrell⁵  Liz Gill⁵ 

[1] Alnylam Pharmaceuticals, Alnylam UK Ltd, Braywick Gate, SL6 1DA, Maidenhead, UK;Alnylam Pharmaceuticals, Cambridge, MA, USA;BresMed Health Solutions Ltd, Steele City House, Sheffield, UK;BresMed Netherlands B.V, HNK Utrecht CS, Utrecht, Netherlands;British Porphyria Association, Durham, UK;

关键词: Porphyria;    Acute hepatic porphyria;    Qualitative;    Quantitative;    Quality of life;    Caregiver experience;    Burden of illness;    Pain;    Chronic symptoms;    Neuropathy;   

DOI  :  10.1186/s13023-021-01816-2

来源: Springer

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