✦ LIBER ✦

154. Disease stability in patients with Niemann–Pick disease type C treated with miglustat

✍ Scribed by Ed Wraith; Darlenn Vecchio; Elizabeth Jacklin; Cicile Luzy; Ruben Giorgino; Marc Patterson

Book ID: 116988588
Publisher: Elsevier Science
Year: 2009
Tongue: English
Weight: 41 KB
Volume: 96
Category: Article
ISSN: 1096-7192
DOI: 10.1016/j.ymgme.2008.11.155

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

112. Safety monitoring of miglustat in p

112. Safety monitoring of miglustat in patients with Niemann-Pick disease type C

✍ Ed Wraith; Michael Beck; Jackie Imrie; Merce Pineda; Barbara Schwierin; Bruno Be 📂 Article 📅 2008 🏛 Elsevier Science 🌐 English ⚖ 43 KB

111. Miglustat in Niemann-Pick disease t

111. Miglustat in Niemann-Pick disease type C (NPC)

✍ Ed Wraith; Darleen Vecchio; Helena Prady; Larry Abel; Marc Patterson 📂 Article 📅 2008 🏛 Elsevier Science 🌐 English ⚖ 43 KB

Long-term efficacy of miglustat in paedi

Long-term efficacy of miglustat in paediatric patients with Niemann-Pick disease type C

✍ Chien, Y. H.; Peng, S. F.; Yang, C. C.; Lee, N. C.; Tsai, L. K.; Huang, A. C.; S 📂 Article 📅 2012 🏛 Springer 🌐 English ⚖ 311 KB

Early Miglustat Therapy in Infantile Nie

Early Miglustat Therapy in Infantile Niemann-Pick Disease Type C

✍ Maja Di Rocco; Andrea Dardis; Annalisa Madeo; Rita Barone; Agata Fiumara 📂 Article 📅 2012 🏛 Elsevier Science 🌐 English ⚖ 107 KB

O6-2 Cerebral PET and disability evaluat

O6-2 Cerebral PET and disability evaluation in patients with Niemann Pick disease Type C treated with miglustat

✍ M.S. Perez Poyato; M. Pineda Marfa; M.A. Vilaseca; M.M. O'Callaghan Gordo; J. Ma 📂 Article 📅 2009 🏛 Elsevier Science 🌐 English ⚖ 43 KB