Individual Treatment Trials-Do Experts Know and Use This Option to Improve the Treatability of Mucopolysaccharidosis?

Wiesinger, Anna-Maria; Strobl, Hannah; Lagler, Florian B

Wiesinger, Anna-Maria; Strobl, Hannah; Lagler, Florian B.

Afiliação

Wiesinger AM; Department of Medical Science, Institute of Congenital Metabolic Diseases, Paracelsus Medical University, 5020 Salzburg, Austria.
Strobl H; Department of Rare Diseases, European Reference Network for Hereditary Metabolic Diseases, MetabERN, 33100 Udine, Italy.
Lagler FB; Department of Medical Science, Institute of Congenital Metabolic Diseases, Paracelsus Medical University, 5020 Salzburg, Austria.

Pharmaceuticals (Basel) ; 16(3)2023 Mar 09.

Article em En | MEDLINE | ID: mdl-36986515

Palavras-chave

ESITT; MPS; decision analysis framework model; expert survey; individual treatment trials; mucopolysaccharidosis; n-of-1; precision medicine; rare disease treatment

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Texto completo: 1 Bases de dados: MEDLINE Tipo de estudo: Prognostic_studies Idioma: En Revista: Pharmaceuticals (Basel) Ano de publicação: 2023 Tipo de documento: Article País de afiliação: Áustria

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