The risks of therapeutic misconception and individual patient (n=1) “trials” in rare diseases such as Duchenne dystrophy

SID > Fuentes Documentales > Publicaciones Periódicas > Artículos de Revistas > The risks of therapeutic misconception and individual patient (n=1) “trials” in rare diseases such as Duchenne dystrophy

Resumen

There is currently no cure for Duchenne muscular dystrophy, but there are some promising treatments in development, of which antisense-mediated exon skipping is close to clinical application. The results of the first trials have resulted in significant enthusiasm among clinicians, patients, and their parents and attracted widespread attention in both the scientific and lay press. As in many other rare diseases with significant morbidity this fuels the hope that at last an effective therapy might be within our reach. Parents, but also some clinicians, are now seriously considering treating individual patients with antisense oligonucleotides without waiting for proof from well-designed clinical trials that the new therapies are indeed effective and safe.

Resumen realizado por los autores recogido de la publicación

Publicación

Reino Unido:
Neuromuscular Disorders: NMD,
2011

ISBN

1873-2364

Descripción física

13-15

Serie

Quizás te interese:

Recibe más documentos en tu email

No te pierdas todas nuestras actualizaciones