Methodology of clinical research in rare diseases: development of a research program in juvenile neuronal ceroid lipofuscinosis (JNCL) via creation of a patient registry and collaboration with patient advocates.
Contemp Clin Trials
; 35(2): 48-54, 2013 Jul.
Article
en En
| MEDLINE
| ID: mdl-23628560
Palabras clave
BDSRA; Batten Disease Support and Research Association; Clinical trials; JNCL; Juvenile neuronal ceroid lipofuscinosis; NCL; NIH; National Institutes of Health; Patient advocacy; Patient registry; Rare disease; Subject recruitment; UBDRS; URBC; Unified Batten Disease Rating Scale; University of Rochester Batten Center; juvenile neuronal ceroid lipofuscinosis; neuronal ceroid lipofuscinosis
Texto completo:
1
Colección:
01-internacional
Base de datos:
MEDLINE
Asunto principal:
Sistema de Registros
/
Ensayos Clínicos como Asunto
/
Selección de Paciente
/
Enfermedades Raras
/
Investigación Biomédica
/
Lipofuscinosis Ceroideas Neuronales
Límite:
Humans
Idioma:
En
Revista:
Contemp Clin Trials
Asunto de la revista:
MEDICINA
/
TERAPEUTICA
Año:
2013
Tipo del documento:
Article
País de afiliación:
Estados Unidos
