| 000 | 01768 a2200493 4500 | ||
|---|---|---|---|
| 005 | 20250518084816.0 | ||
| 264 | 0 | _c20201123 | |
| 008 | 202011s 0 0 eng d | ||
| 022 | _a1096-7206 | ||
| 024 | 7 |
_a10.1016/j.ymgme.2020.01.003 _2doi |
|
| 040 |
_aNLM _beng _cNLM |
||
| 100 | 1 | _aWang, Raymond Y | |
| 245 | 0 | 0 |
_aThe long-term safety and efficacy of vestronidase alfa, rhGUS enzyme replacement therapy, in subjects with mucopolysaccharidosis VII. _h[electronic resource] |
| 260 |
_bMolecular genetics and metabolism _c03 2020 |
||
| 300 |
_a219-227 p. _bdigital |
||
| 500 | _aPublication Type: Clinical Trial, Phase III; Journal Article; Randomized Controlled Trial; Research Support, Non-U.S. Gov't | ||
| 650 | 0 | 4 | _aAdolescent |
| 650 | 0 | 4 | _aAdult |
| 650 | 0 | 4 | _aAntibodies, Neutralizing |
| 650 | 0 | 4 |
_aBlood-Brain Barrier _xdrug effects |
| 650 | 0 | 4 | _aChild |
| 650 | 0 | 4 | _aCross-Over Studies |
| 650 | 0 | 4 | _aEnzyme Replacement Therapy |
| 650 | 0 | 4 | _aFemale |
| 650 | 0 | 4 |
_aGlucuronidase _xadministration & dosage |
| 650 | 0 | 4 |
_aGlycosaminoglycans _xurine |
| 650 | 0 | 4 | _aHumans |
| 650 | 0 | 4 | _aMale |
| 650 | 0 | 4 |
_aMucopolysaccharidosis VII _xdrug therapy |
| 650 | 0 | 4 |
_aRare Diseases _xtherapy |
| 650 | 0 | 4 | _aTreatment Outcome |
| 700 | 1 | _ada Silva Franco, José Francisco | |
| 700 | 1 | _aLópez-Valdez, Jaime | |
| 700 | 1 | _aMartins, Esmeralda | |
| 700 | 1 | _aSutton, Vernon Reid | |
| 700 | 1 | _aWhitley, Chester B | |
| 700 | 1 | _aZhang, Lin | |
| 700 | 1 | _aCimms, Tricia | |
| 700 | 1 | _aMarsden, Deborah | |
| 700 | 1 | _aJurecka, Agnieszka | |
| 700 | 1 | _aHarmatz, Paul | |
| 773 | 0 |
_tMolecular genetics and metabolism _gvol. 129 _gno. 3 _gp. 219-227 |
|
| 856 | 4 | 0 |
_uhttps://doi.org/10.1016/j.ymgme.2020.01.003 _zAvailable from publisher's website |
| 999 |
_c30639791 _d30639791 |
||