PMID- 34424497
OWN - NLM
STAT- MEDLINE
DCOM- 20220422
LR  - 20220422
IS  - 1179-2000 (Electronic)
IS  - 1177-1062 (Linking)
VI  - 25
IP  - 6
DP  - 2021 Nov
TI  - Elivaldogene Autotemcel: First Approval.
PG  - 803-809
LID - 10.1007/s40291-021-00555-1 [doi]
AB  - Elivaldogene autotemcel (SKYSONA, eli-cel; Lenti-D gene therapy) is a gene 
      therapy that has been developed by bluebird bio for the treatment of cerebral 
      adrenoleukodystrophy (CALD), a rare, X-linked genetic disease that mainly affects 
      the nervous system and adrenal glands. In July 2021, elivaldogene autotemcel 
      received its first approval (in the EU) for the treatment of for the treatment of 
      early CALD in patients < 18 years of age, with an ABCD1 genetic mutation, and for 
      whom a human leukocyte antigen (HLA)-matched sibling haematopoietic stem cell 
      (HSC) donor is not available. This article summarizes the milestones in the 
      development of elivaldogene autotemcel leading to this first approval.
CI  - (c) 2021. The Author(s), under exclusive licence to Springer Nature Switzerland AG.
FAU - Keam, Susan J
AU  - Keam SJ
AD  - Springer Nature, Mairangi Bay, Private Bag 65901, Auckland, 0754, New Zealand. 
      mdt@adis.com.
LA  - eng
SI  - Dryad/10.6084/m9.figshare.15161709
PT  - Journal Article
PT  - Review
DEP - 20210823
PL  - New Zealand
TA  - Mol Diagn Ther
JT  - Molecular diagnosis & therapy
JID - 101264260
SB  - IM
MH  - *Adrenoleukodystrophy/genetics/therapy
MH  - Genetic Therapy
MH  - Humans
EDAT- 2021/08/24 06:00
MHDA- 2022/04/23 06:00
CRDT- 2021/08/23 12:32
PHST- 2021/08/13 00:00 [accepted]
PHST- 2021/08/24 06:00 [pubmed]
PHST- 2022/04/23 06:00 [medline]
PHST- 2021/08/23 12:32 [entrez]
AID - 10.1007/s40291-021-00555-1 [pii]
AID - 10.1007/s40291-021-00555-1 [doi]
PST - ppublish
SO  - Mol Diagn Ther. 2021 Nov;25(6):803-809. doi: 10.1007/s40291-021-00555-1. Epub 
      2021 Aug 23.