| 000 | 01602 a2200445 4500 | ||
|---|---|---|---|
| 005 | 20250517033909.0 | ||
| 264 | 0 | _c20160229 | |
| 008 | 201602s 0 0 eng d | ||
| 022 | _a1750-1172 | ||
| 024 | 7 |
_a10.1186/s13023-015-0284-z _2doi |
|
| 040 |
_aNLM _beng _cNLM |
||
| 100 | 1 | _aPatterson, Marc C | |
| 245 | 0 | 0 |
_aStable or improved neurological manifestations during miglustat therapy in patients from the international disease registry for Niemann-Pick disease type C: an observational cohort study. _h[electronic resource] |
| 260 |
_bOrphanet journal of rare diseases _cMay 2015 |
||
| 300 |
_a65 p. _bdigital |
||
| 500 | _aPublication Type: Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't | ||
| 650 | 0 | 4 |
_a1-Deoxynojirimycin _xanalogs & derivatives |
| 650 | 0 | 4 | _aAdolescent |
| 650 | 0 | 4 | _aAdult |
| 650 | 0 | 4 | _aChild |
| 650 | 0 | 4 | _aChild, Preschool |
| 650 | 0 | 4 | _aCohort Studies |
| 650 | 0 | 4 | _aDisease Progression |
| 650 | 0 | 4 |
_aEnzyme Inhibitors _xtherapeutic use |
| 650 | 0 | 4 | _aFemale |
| 650 | 0 | 4 | _aHumans |
| 650 | 0 | 4 | _aMale |
| 650 | 0 | 4 |
_aNiemann-Pick Disease, Type C _xdrug therapy |
| 650 | 0 | 4 | _aProspective Studies |
| 650 | 0 | 4 | _aTreatment Outcome |
| 650 | 0 | 4 | _aYoung Adult |
| 700 | 1 | _aMengel, Eugen | |
| 700 | 1 | _aVanier, Marie T | |
| 700 | 1 | _aSchwierin, Barbara | |
| 700 | 1 | _aMuller, Audrey | |
| 700 | 1 | _aCornelisse, Peter | |
| 700 | 1 | _aPineda, Mercè | |
| 773 | 0 |
_tOrphanet journal of rare diseases _gvol. 10 _gp. 65 |
|
| 856 | 4 | 0 |
_uhttps://doi.org/10.1186/s13023-015-0284-z _zAvailable from publisher's website |
| 999 |
_c24939586 _d24939586 |
||