| 000 | 01504 a2200409 4500 | ||
|---|---|---|---|
| 005 | 20250517202826.0 | ||
| 264 | 0 | _c20190708 | |
| 008 | 201907s 0 0 eng d | ||
| 022 | _a1096-7206 | ||
| 024 | 7 |
_a10.1016/j.ymgme.2018.02.006 _2doi |
|
| 040 |
_aNLM _beng _cNLM |
||
| 100 | 1 | _aHarmatz, Paul | |
| 245 | 0 | 0 |
_aA novel Blind Start study design to investigate vestronidase alfa for mucopolysaccharidosis VII, an ultra-rare genetic disease. _h[electronic resource] |
| 260 |
_bMolecular genetics and metabolism _c04 2018 |
||
| 300 |
_a488-494 p. _bdigital |
||
| 500 | _aPublication Type: Clinical Trial, Phase III; Journal Article; Randomized Controlled Trial; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't | ||
| 650 | 0 | 4 | _aAdolescent |
| 650 | 0 | 4 | _aAdult |
| 650 | 0 | 4 | _aChild |
| 650 | 0 | 4 | _aFemale |
| 650 | 0 | 4 | _aFollow-Up Studies |
| 650 | 0 | 4 |
_aGlucuronidase _xadministration & dosage |
| 650 | 0 | 4 | _aHumans |
| 650 | 0 | 4 | _aMale |
| 650 | 0 | 4 |
_aMucopolysaccharidosis VII _xenzymology |
| 650 | 0 | 4 | _aPrognosis |
| 650 | 0 | 4 |
_aRecombinant Proteins _xadministration & dosage |
| 650 | 0 | 4 | _aYoung Adult |
| 700 | 1 | _aWhitley, Chester B | |
| 700 | 1 | _aWang, Raymond Y | |
| 700 | 1 | _aBauer, Mislen | |
| 700 | 1 | _aSong, Wenjie | |
| 700 | 1 | _aHaller, Christine | |
| 700 | 1 | _aKakkis, Emil | |
| 773 | 0 |
_tMolecular genetics and metabolism _gvol. 123 _gno. 4 _gp. 488-494 |
|
| 856 | 4 | 0 |
_uhttps://doi.org/10.1016/j.ymgme.2018.02.006 _zAvailable from publisher's website |
| 999 |
_c28120535 _d28120535 |
||