Implications of circulating neurofilaments for spinal muscular atrophy treatment early in life: A case series.
Biomarkers
Gene Therapy
Neurogenetics
Neuromuscular diseases
SMA
Journal
Molecular therapy. Methods & clinical development
ISSN: 2329-0501
Titre abrégé: Mol Ther Methods Clin Dev
Pays: United States
ID NLM: 101624857
Informations de publication
Date de publication:
10 Dec 2021
10 Dec 2021
Historique:
received:
22
08
2021
revised:
06
10
2021
accepted:
26
10
2021
pubmed:
3
12
2021
medline:
3
12
2021
entrez:
2
12
2021
Statut:
epublish
Résumé
This longitudinal cohort study aimed to determine whether circulating neurofilaments (NFs) can monitor response to molecular therapies in newborns with spinal muscular atrophy (SMA; NCT02831296). We applied a mixed-effect model to examine differences in serum NF levels among healthy control infants (n = 13), untreated SMA infants (n = 68), and SMA infants who received the genetic therapies nusinersen and/or onasemnogene abeparvovec (n = 22). Increased NF levels were inversely associated with
Identifiants
pubmed: 34853799
doi: 10.1016/j.omtm.2021.10.011
pii: S2329-0501(21)00168-6
pmc: PMC8605296
doi:
Types de publication
Journal Article
Langues
eng
Pagination
524-538Subventions
Organisme : NICHD NIH HHS
ID : R01 HD054599
Pays : United States
Organisme : NINDS NIH HHS
ID : R21 NS108015
Pays : United States
Informations de copyright
© 2021 The Authors.
Déclaration de conflit d'intérêts
C.R.R.A. and K.J.S. are inventors on a patent filed by Mass General Brigham that describes genome engineering technologies to treat SMA. C.R.R.A. holds stocks in CRISPR Therapeutics. K.J.S. is the recipient of a grant from Biogen and receives clinical trial funding from AveXis and Biogen. E.L.T. serves on a physical therapy advisory board for Biogen. M.P., J.S., and W.F. are current or former employees of Biogen and hold stock/stock options in Biogen. R.S., R.G., R.Z., E.J.E., E.R., E.A.A., S.S., and M.K. report no conflict of interest.