Exon skipping in Duchenne muscular dystrophy
Metadatos
Mostrar el registro completo del ítemcomunitat-uji-handle:10234/158176
comunitat-uji-handle2:10234/71324
comunitat-uji-handle3:10234/167590
comunitat-uji-handle4:
TFG-TFMEste recurso está restringido
Metadatos
Título
Exon skipping in Duchenne muscular dystrophyAutoría
Tutor/Supervisor; Universidad.Departamento
Sánchez Pérez, Ana Maria; Universitat Jaume I. Unitat Predepartamental de Medicina.Fecha de publicación
2020-04-28Editor
Universitat Jaume IResumen
Duchenne muscular dystrophy (DMD) is the most common form of muscular dystrophy.
Current treatment for this disease focuses on a symptomatic approach, however,
mutation-specific therapies have gained regulatory ... [+]
Duchenne muscular dystrophy (DMD) is the most common form of muscular dystrophy.
Current treatment for this disease focuses on a symptomatic approach, however,
mutation-specific therapies have gained regulatory approval, offering the potential of a
long-term correction.
This systematic review’s aim is to compare the efficacy and safety profile of exon
skipping therapy for DMD. To achieve this, 5 clinical trials were analyzed on two exon
skipping therapies, selected after a structured Pubmed and Embase search; eteplirsen
(an already approved treatment) and drisapersen. For this purpose, the variables of
motor function, and adverse events were studied, among others.
To evaluate the methodological quality, the Cochrane Manual of Systematic
Reviews (version 5.1.0) was used, as well as the Scottish Intercollegiate Guidelines
Network (SIGN).
Both therapies had limitations, for example, eteplirsen studies displayed a
moderate risk of bias and drisapersen had safety issues that caused its rejection by the
FDA. Even though they both showed clinical benefits in the right subgroups of population
and demonstrated to improve the survival and motor function of DMD patients, the
heterogenicity between the samples and design of the studies of both therapies prevents
a well-founded comparison. [-]
Palabras clave / Materias
Descripción
Treball Final de Grau en Medicina. Codi: MD1158. Curs acadèmic: 2019/2020
Tipo de documento
info:eu-repo/semantics/bachelorThesisDerechos de acceso
http://rightsstatements.org/vocab/CNE/1.0/
info:eu-repo/semantics/restrictedAccess
info:eu-repo/semantics/restrictedAccess
Aparece en las colecciones
- Grau en Medicina [495]