Cargando…
Effect of enzyme therapy and prognostic factors in 69 adults with Pompe disease: an open-label single-center study
BACKGROUND: Enzyme replacement therapy (ERT) in adults with Pompe disease, a progressive neuromuscular disorder, is of promising but variable efficacy. We investigated whether it alters the course of disease, and also identified potential prognostic factors. METHODS: Patients in this open-label sing...
Autores principales: | , , , , , , , , , , , , , , , |
---|---|
Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
BioMed Central
2012
|
Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3519647/ https://www.ncbi.nlm.nih.gov/pubmed/23013746 http://dx.doi.org/10.1186/1750-1172-7-73 |