Long term follow-up to evaluate the efficacy of miglustat treatment in Italian patients with Niemann-Pick disease type C

Efficacy of Miglustat in Niemann–Pick C disease: A single centre experience

(2013) Virginia Maria Ginocchio et al.   MOLECULAR GENETICS AND METABOLISM

Effects of Miglustat on Stabilization of Neurological Disorder in Niemann–Pick Disease Type C

(2012) Parvaneh Karimzadeh et al.   JOURNAL OF CHILD NEUROLOGY

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

(2012) Y. H. Chien et al.   JOURNAL OF INHERITED METABOLIC DISEASE

Recommendations for the diagnosis and management of Niemann–Pick disease type C: An update

(2012) Marc C. Patterson et al.   MOLECULAR GENETICS AND METABOLISM

Miglustat therapy in the French cohort of paediatric patients with Niemann-Pick disease type C

(2012) Bénédicte Héron et al.   Orphanet Journal of Rare Diseases

Dysphagia as a risk factor for mortality in Niemann-Pick disease type C: systematic literature review and evidence from studies with miglustat

(2012) Mark Walterfang et al.   Orphanet Journal of Rare Diseases

Early Miglustat Therapy in Infantile Niemann-Pick Disease Type C

(2012) Maja Di Rocco et al.   PEDIATRIC NEUROLOGY

The videofluoroscopic swallowing study shows a sustained improvement of dysphagia in children with Niemann-Pick disease type C after therapy with miglustat

(2011) Simona Fecarotta et al.   AMERICAN JOURNAL OF MEDICAL GENETICS PART A

Niemann-Pick disease type C

(2010) Marie T Vanier   Orphanet Journal of Rare Diseases

Long-Term Miglustat Therapy in Children With Niemann-Pick Disease Type C

(2009) Marc C. Patterson et al.   JOURNAL OF CHILD NEUROLOGY

Miglustat in patients with Niemann-Pick disease Type C (NP-C): A multicenter observational retrospective cohort study

(2009) M. Pineda et al.   MOLECULAR GENETICS AND METABOLISM

Natural history of Niemann-Pick disease type C in a multicentre observational retrospective cohort study

(2009) J.E. Wraith et al.   MOLECULAR GENETICS AND METABOLISM

Clinical experience with miglustat therapy in pediatric patients with Niemann–Pick disease type C: A case series

(2009) M. Pineda et al.   MOLECULAR GENETICS AND METABOLISM

Treatment of a child diagnosed with Niemann–Pick disease type C with miglustat: A case report in Brazil

(2008) M. L. F. Santos et al.   JOURNAL OF INHERITED METABOLIC DISEASE