Disease characteristics, prognosis and miglustat treatment effects on disease progression in patients with Niemann-Pick disease Type C: an international, multicenter, retrospective chart review.
Disease characteristics, prognosis and miglustat treatment effects on disease progression in patients with Niemann-Pick disease Type C: an international, multicenter, retrospective chart review. — المؤلف: Pineda, Mercedes — Orphanet journal of rare diseases 20190419
- المؤلف
- Pineda, Mercedes
- النشر
- Orphanet journal of rare diseases 20190419
- الوصف
- 32 p.
- الموضوعات
- 1-Deoxynojirimycin؛ Adolescent؛ Child؛ Child, Preschool؛ Female؛ Humans؛ Linear Models؛ Male