Antisense-based therapy for the treatment of spinal muscular atrophy.
J Cell Biol
; 199(1): 21-5, 2012 Oct 01.
Article
em En
| MEDLINE
| ID: mdl-23027901
ABSTRACT
One of the greatest thrills a biomedical researcher may experience is seeing the product of many years of dedicated effort finally make its way to the patient. As a team, we have worked for the past eight years to discover a drug that could treat a devastating childhood neuromuscular disease, spinal muscular atrophy (SMA). Here, we describe the journey that has led to a promising drug based on the biology underlying the disease.
Texto completo:
1
Base de dados:
MEDLINE
Assunto principal:
Atrofia Muscular Espinal
/
Oligonucleotídeos Antissenso
Idioma:
En
Ano de publicação:
2012
Tipo de documento:
Article