000			01988 a2200601 4500
005			20250516094804.0
264		0	_c20130103
008			201301s 0 0 eng d
022			_a1750-1172
024	7		_a10.1186/1750-1172-7-36 _2doi
040			_aNLM _beng _cNLM
100	1		_aHéron, Bénédicte
245	0	0	_aMiglustat therapy in the French cohort of paediatric patients with Niemann-Pick disease type C. _h[electronic resource]
260			_bOrphanet journal of rare diseases _cJun 2012
300			_a36 p. _bdigital
500			_aPublication Type: Clinical Trial; Journal Article
650	0	4	_a1-Deoxynojirimycin _xadministration & dosage
650	0	4	_aAdolescent
650	0	4	_aAge of Onset
650	0	4	_aCarrier Proteins _xgenetics
650	0	4	_aChild
650	0	4	_aChild, Preschool
650	0	4	_aCohort Studies
650	0	4	_aEnzyme Inhibitors _xadministration & dosage
650	0	4	_aFemale
650	0	4	_aFrance
650	0	4	_aGlycoproteins _xgenetics
650	0	4	_aHumans
650	0	4	_aInfant
650	0	4	_aIntracellular Signaling Peptides and Proteins
650	0	4	_aMale
650	0	4	_aMembrane Glycoproteins _xgenetics
650	0	4	_aNiemann-Pick C1 Protein
650	0	4	_aNiemann-Pick Disease, Type C _xdrug therapy
650	0	4	_aProspective Studies
650	0	4	_aTreatment Outcome
650	0	4	_aVesicular Transport Proteins
700	1		_aValayannopoulos, Vassili
700	1		_aBaruteau, Julien
700	1		_aChabrol, Brigitte
700	1		_aOgier, Hélène
700	1		_aLatour, Philippe
700	1		_aDobbelaere, Dries
700	1		_aEyer, Didier
700	1		_aLabarthe, François
700	1		_aMaurey, Hélène
700	1		_aCuisset, Jean-Marie
700	1		_ade Villemeur, Thierry Billette
700	1		_aSedel, Frédéric
700	1		_aVanier, Marie T
773	0		_tOrphanet journal of rare diseases _gvol. 7 _gp. 36
856	4	0	_uhttps://doi.org/10.1186/1750-1172-7-36 _zAvailable from publisher's website
999			_c21845696 _d21845696