Clinically meaningful improvements in patient-reported outcomes in mitapivat-treated patients with pyruvate kinase deficiency.
Am J Hematol
; 99(7): 1415-1419, 2024 07.
Article
em En
| MEDLINE
| ID: mdl-38712615
ABSTRACT
Clinically meaningful benefits in the signs, symptoms, and impacts of #PKDeficiency as assessed by disease-specific patient-reported outcome measures were observed in mitapivat-treated adult patients in two phase 3 clinical trials.
Texto completo:
1
Base de dados:
MEDLINE
Assunto principal:
Piruvato Quinase
/
Erros Inatos do Metabolismo dos Piruvatos
/
Medidas de Resultados Relatados pelo Paciente
Idioma:
En
Ano de publicação:
2024
Tipo de documento:
Article