Treatment with Nusinersen - Challenges Regarding the Indication for Children with SMA Type 1.
J Neuromuscul Dis
; 7(1): 41-46, 2020.
Article
en En
| MEDLINE
| ID: mdl-31744015
ABSTRACT
The natural history of patients with spinal muscular atrophy (SMA) has changed due to advances in standard care and development of targeted treatments. Nusinersen was the first drug approved for the treatment of all SMA patients. The transfer of clinical trial data into a real-life environment is challenging, especially regarding the advice of patients and families to what extent they can expect a benefit from the novel treatment. We report the results of a modified Delphi consensus process among child neurologists from Germany, Austria and Switzerland about the indication or continuation of nusinersen treatment in children with SMA type 1 based on different clinical case scenarios.
Texto completo:
1
Banco de datos:
MEDLINE
Asunto principal:
Oligonucleótidos
/
Atrofias Musculares Espinales de la Infancia
/
Consenso
/
Neurólogos
/
Pediatras
Tipo de estudio:
Guideline
Límite:
Child
/
Humans
País/Región como asunto:
Europa
Idioma:
En
Año:
2020
Tipo del documento:
Article