Miglustat therapy in the French cohort of paediatric patients with Niemann-Pick disease type C. [electronic resource]
Producer: 20130103Description: 36 p. digitalISSN:- 1750-1172
- 1-Deoxynojirimycin -- administration & dosage
- Adolescent
- Age of Onset
- Carrier Proteins -- genetics
- Child
- Child, Preschool
- Cohort Studies
- Enzyme Inhibitors -- administration & dosage
- Female
- France
- Glycoproteins -- genetics
- Humans
- Infant
- Intracellular Signaling Peptides and Proteins
- Male
- Membrane Glycoproteins -- genetics
- Niemann-Pick C1 Protein
- Niemann-Pick Disease, Type C -- drug therapy
- Prospective Studies
- Treatment Outcome
- Vesicular Transport Proteins
No physical items for this record
Publication Type: Clinical Trial; Journal Article
There are no comments on this title.
Log in to your account to post a comment.