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Understanding the challenges, unmet needs, and expectations of mucopolysaccharidoses I, II and VI patients and their caregivers in France: a survey study.
Guffon, Nathalie; Genevaz, Delphine; Lacombe, Didier; Le Peillet Feuillet, Eliane; Bausson, Pascale; Noel, Esther; Maillot, François; Belmatoug, Nadia; Jaussaud, Roland.
Afiliação
  • Guffon N; Reference Center for Inherited Metabolic Disorders of Lyon, (CERLYMM), Hospices Civils de Lyon, 69677, Bron, France. nathalie.guffon-fouilhoux@chu-lyon.fr.
  • Genevaz D; Vaincre Les Maladies Lysosomales, 91300, Massy, France.
  • Lacombe D; Medical Genetics Unit, University Hospital of Bordeaux, INSERM U1211, 33076, Bordeaux, France.
  • Le Peillet Feuillet E; Sanofi, 94250, Gentilly, France.
  • Bausson P; Study Department, AplusA Company, 92641, Boulogne Billancourt, France.
  • Noel E; University Hospital of Strasbourg, BP 426, 67100, Strasbourg, France.
  • Maillot F; Department of Internal Medicine, Regional University Hospital of Tours, 37000, Tours, France.
  • Belmatoug N; Reference Center of Lysosomal Diseases, Beaujon Hospital, 92110, Clichy, France.
  • Jaussaud R; Department of Internal Medicine and Clinical Immunology, Nancy University Hospital, 54500, Vandoeuvre-Les-Nancy, France.
Orphanet J Rare Dis ; 17(1): 448, 2022 12 23.
Article em En | MEDLINE | ID: mdl-36564803

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Mucopolissacaridose I / Mucopolissacaridoses / Pessoas com Deficiência / Transtornos Motores Idioma: En Ano de publicação: 2022 Tipo de documento: Article

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Mucopolissacaridose I / Mucopolissacaridoses / Pessoas com Deficiência / Transtornos Motores Idioma: En Ano de publicação: 2022 Tipo de documento: Article