Zobrazeno 1 - 3
of 3
pro vyhledávání: '"Natalie L. Goedeker"'
Autor:
Natalie L. Goedeker, Sachi D. Dharia, Danielle A. Griffin, Jesantha Coy, Todd Truesdale, Rajan Parikh, Kasen Whitehouse, Sourav Santra, Damon R. Asher, Craig M. Zaidman
Publikováno v:
Therapeutic Advances in Neurological Disorders, Vol 16 (2023)
Background: Adeno-associated virus (AAV) vectors are a promising platform for in vivo transfer of transgenes designed to treat diseases. Pre-existing humoral immunity to these vectors can potentially impact the safety and efficacy of gene therapies.
Externí odkaz:
https://doaj.org/article/3188b9979c4a41868a6b7e86441ae1d1
Autor:
Kapil Arya, Deepa H. Chand, Vikas R. Dharnidharka, Michelle A. Farrar, Sandra P. Reyna, Natalie L. Goedeker, Raja Dandamudi, Fiona E. Mackie, Craig M. Zaidman, Rachel Millner
Publikováno v:
The Journal of Pediatrics. 231:265-268
Spinal muscular atrophy is treated with onasemnogene abeparvovec, which replaces the missing survival motor neuron 1 gene via an adeno-associated virus vector. As of July 1, 2020, we had identified 3 infants who developed thrombotic microangiopathy f
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::66a127dffdaff1a0becd20a845571963
https://doi.org/10.1016/j.jpeds.2020.11.054
https://doi.org/10.1016/j.jpeds.2020.11.054
Autor:
Craig M. Zaidman, Arun S. Varadhachary, Anne M. Connolly, Jennifer L Gibbons, Natalie L. Goedeker
Publikováno v:
Muscle & Nerve. 63:902-905
Introduction This retrospective study reports our tertiary care center's experience with intrathecal nusinersen administration in children and adults with spinal muscular atrophy (SMA). Methods We reviewed safety monitoring laboratory results and nee
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::cb82b85df2f07e4f9da6422b14a03a46
https://doi.org/10.1002/mus.27217
https://doi.org/10.1002/mus.27217