Interview-Based Patient- and Caregiver-Reported Experiences of Hunger and Improved Quality of Life with Setmelanotide Treatment in Bardet-Biedl Syndrome.

Ervin, Claire; Norcross, Lindsey; Mallya, Usha G; Fehnel, Sheri; Mittleman, Robert S; Webster, Matthew; Haqq, Andrea M; Haws, Robert M

Ervin, Claire; Norcross, Lindsey; Mallya, Usha G; Fehnel, Sheri; Mittleman, Robert S; Webster, Matthew; Haqq, Andrea M; Haws, Robert M.

Afiliação

Ervin C; RTI Health Solutions, 3040 E Cornwallis Rd, PO Box 12194, Research Triangle Park, NC, 27709-2194, USA. cervin@rti.org.
Norcross L; RTI Health Solutions, 3040 E Cornwallis Rd, PO Box 12194, Research Triangle Park, NC, 27709-2194, USA.
Mallya UG; Rhythm Pharmaceuticals, Inc., 222 Berkeley Street, 12th Floor, Boston, MA, 02116, USA.
Fehnel S; RTI Health Solutions, 3040 E Cornwallis Rd, PO Box 12194, Research Triangle Park, NC, 27709-2194, USA.
Mittleman RS; Rhythm Pharmaceuticals, Inc., 222 Berkeley Street, 12th Floor, Boston, MA, 02116, USA.
Webster M; Rhythm Pharmaceuticals, Inc., 222 Berkeley Street, 12th Floor, Boston, MA, 02116, USA.
Haqq AM; University of Alberta, 6-002E Li Ka Shing Centre for Health Research Innovation, Edmonton, AB, T6G 2E1, Canada.
Haws RM; Marshfield Clinic Research Institute, 1000 North Oak Avenue, Marshfield, WI, 54449, USA.

Adv Ther ; 40(5): 2394-2411, 2023 05.

Article em En | MEDLINE | ID: mdl-36961653

Assuntos

Síndrome de Bardet-Biedl; Qualidade de Vida; Criança; Humanos; Cuidadores/psicologia; Síndrome de Bardet-Biedl/tratamento farmacológico; alfa-MSH/uso terapêutico

Palavras-chave

Bardet-Biedl syndrome; Disease burden; Hyperphagia; MC4R pathway; Quality of life; Setmelanotide

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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Qualidade de Vida / Síndrome de Bardet-Biedl Tipo de estudo: Qualitative_research Limite: Child / Humans Idioma: En Ano de publicação: 2023 Tipo de documento: Article

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