Establishing the appropriate primary endpoint in haemophilia gene therapy pivotal studies.

Pierce, G F; Ragni, M V; van den Berg, H M; Weill, A; O'Mahony, B; Skinner, M W; Pipe, S W

Pierce, G F; Ragni, M V; van den Berg, H M; Weill, A; O'Mahony, B; Skinner, M W; Pipe, S W.

Afiliação

Pierce GF; National Hemophilia Foundation Medical and Scientific Advisory Council, New York, NY, USA.
Ragni MV; World Federation of Hemophilia, Montreal, Quebec, Canada.
van den Berg HM; National Hemophilia Foundation Medical and Scientific Advisory Council, New York, NY, USA.
Weill A; World Federation of Hemophilia, Montreal, Quebec, Canada.
O'Mahony B; World Federation of Hemophilia, Montreal, Quebec, Canada.
Skinner MW; European Haemophilia Consortium, Brussels, Belgium.
Pipe SW; National Hemophilia Foundation Medical and Scientific Advisory Council, New York, NY, USA.

Haemophilia ; 23(5): 643-644, 2017 09.

Article em En | MEDLINE | ID: mdl-28833947

Assuntos

Determinação de Ponto Final/métodos; Terapia Genética; Hemofilia A/genética; Hemofilia A/terapia; Humanos

Palavras-chave

annualized bleeding rate; clinical trials; factor IX; factor VIII; gene therapy; haemophilia

Texto completo

Imprimir

XML

PubMed Links

Buscar no Google

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Terapia Genética / Determinação de Ponto Final / Hemofilia A Limite: Humans Idioma: En Ano de publicação: 2017 Tipo de documento: Article

Texto completo

Imprimir

XML

PubMed Links

Buscar no Google