𝔖 Bobbio Scriptorium
✦   LIBER   ✦

Use of miglustat in a child with late-infantile-onset Niemann-Pick disease type C and frequent seizures: a case report

✍ Scribed by Johannes Skorpen, Ingrid B Helland, Bjørn Tennøe

Book ID
120718287
Publisher
BioMed Central
Year
2012
Tongue
English
Weight
210 KB
Volume
6
Category
Article
ISSN
1752-1947

No coin nor oath required. For personal study only.

📜 SIMILAR VOLUMES

Motion analysis of a child with Niemann–
Motion analysis of a child with Niemann–Pick disease type C treated with miglustat
✍ Alex R. Paciorkowski; Melany Westwell; Sylvia Õunpuu; Katharine Bell; Jeanne Kag 📂 Article 📅 2008 🏛 John Wiley and Sons 🌐 English ⚖ 143 KB

## Abstract Niemann–Pick disease type C (NPC) is a progressive neurodegenerative disorder for which there is no effective treatment other than supportive therapy. Recently, the oral medication miglustat has been offered as a possible therapy aimed at reducing pathological substrate accumulation. Th