Longitudinal Changes of Clinical, Imaging, and Fluid Biomarkers in Preataxic and Early Ataxic Spinocerebellar Ataxia Type 2 and 7 Carriers-Reference-Cited by-同舟云学术

Longitudinal Changes of Clinical, Imaging, and Fluid Biomarkers in Preataxic and Early Ataxic Spinocerebellar Ataxia Type 2 and 7 Carriers

Published:2024-09-10 Issue:5 Volume:103 Page:
ISSN:0028-3878
Container-title:Neurology
language:en
Short-container-title:Neurology

Author:

Coarelli Giulia¹^ORCID,Dubec-Fleury Charlotte¹,Petit Emilien¹,Sayah Sabrina¹,Fischer Clara¹,Nassisi Marco¹,Gatignol Peggy¹,Dorgham Karim¹,Daghsen Lina¹,Daye Pierre¹,Cunha Paulina¹,Kacher Radhia¹,Hilab Rania¹,Hurmic Hortense¹,Lamazière Antonin¹,Lamy Jean-Charles¹,Welter Marie-Laure¹^ORCID,Chupin Marie¹,Mangin Jean-François¹,Lane Roger¹,Gaymard Bertrand¹,Pouget Pierre¹,Audo Isabelle¹,Brice Alexis¹,Tezenas du Montcel Sophie¹^ORCID,Durr Alexandra¹^ORCID

Affiliation:

1. From the Sorbonne Université (G.C., C.D.-F., E.P., S.S., L.D., P.C., R.K., R.H., H.H., J.-C.L., M.-L.W., P.P., A.B., S.T.d.M., A.D.), Paris Brain Institute, Inserm, CNRS, INRIA, APHP; CATI (C.F., M.C., J.-F.M.), US52-UAR2031, CEA, Paris Brain Institute, Sorbonne Université, CNRS, INSERM, APHP; Sorbonne Université (M.N., I.A.), Inserm, CNRS, Institut de la Vision; Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts (M.N., I.A.), National Rare Disease Center REFERET and INSERM-DGOS CIC 1423;...

Publisher

Ovid Technologies (Wolters Kluwer Health)

Link

https://www.neurology.org/doi/pdfdirect/10.1212/WNL.0000000000209749

Reference49 articles.

1. Baseline Clinical and Blood Biomarkers in Patients With Preataxic and Early-Stage Disease Spinocerebellar Ataxia 1 and 3

2. Autosomal dominant cerebellar ataxias: new genes and progress towards treatments

3. Antisense oligonucleotide–mediated ataxin-1 reduction prolongs survival in SCA1 mice and reveals disease-associated transcriptome profiles

4. Antisense oligonucleotide therapy for spinocerebellar ataxia type 2

5. Antisense oligonucleotides targeting mutant Ataxin-7 restore visual function in a mouse model of spinocerebellar ataxia type 7