Mri End-Points for Clinical Trials in Ataxias: Recommendations From the Ataxia Global Initiative Mri Biomarkers Working Group

Mri End-Points for Clinical Trials in Ataxias: Recommendations From the Ataxia Global Initiative Mri Biomarkers Working Group Publisher Pubmed

Oz G ; Cocozza S ; Rezende T J R ; Henry P G ; Faber J ; Harding I H ; Ying S H ; Timmann D ; Thieme A ; Sestakauskas K ; Sarro L ; Santana M M ; Rylski M ; Reetz K Show All Authors

Oz G
Cocozza S
Rezende T J R
Henry P G
Faber J
Harding I H
Ying S H
Timmann D
Thieme A
Sestakauskas K
Sarro L
Santana M M
Rylski M
Reetz K
Ratai E M
Pandolfo M
O Keefe J A
Nobre R J
Nigri A
Nachbauer W
Rangel D M
Mochel F
Minnerop M
Marvel C L
Mariotti C
Makowska L
Lenglet C
Kovacs N
Kinnunen K M
Kapteijns K
Joers J M
Indelicato E
Heidari M
Grisoli M
Gazdzinski S
Fuin N
Franca M C JR
Ferreira M
D Ostilio K
Deistung A
Cossa A
Boesch S
Bando K
Alhusaini S
Adarmes Gomez A
Accolla E A

Source: Nature Reviews Neurology Published:2026

Abstract

Disease-modifying therapies for degenerative ataxias, including emerging gene therapies, are in the clinical trial pipeline. Sensitive outcome measures are urgently needed for treatment monitoring and participant selection to improve trial feasibility in these rare diseases. In hereditary ataxias, the ability to identify people who carry disease-causing mutations before symptom onset also raises the possibility of enrolment into trials before symptom manifestation, but biomarker data are key for participant selection and outcome monitoring in such preventive trials. Quantitative neuroimaging readouts have emerged as viable candidate biomarkers of ataxia pathology and progression that could supplement traditional clinical outcome assessments as clinical trial end-points. In this Consensus Statement, the Ataxia Global Initiative MRI Biomarkers Working Group critically reviews candidate MRI end-points for trials in the most common spinocerebellar ataxias (SCA1, SCA2 and SCA3) and Friedreich ataxia and provides evidence-based, disease-specific recommendations for the selection of MRI end-points for trials in these diseases. Recommendations are also provided for further research to address remaining knowledge gaps. © Springer Nature Limited 2026.

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Style	Citing Format
MLA	Oz G, et al.. "Mri End-Points for Clinical Trials in Ataxias: Recommendations From the Ataxia Global Initiative Mri Biomarkers Working Group." Nature Reviews Neurology, vol. 22, no. 7, 2026, pp. 439-456.
APA	Oz G, Cocozza S, Rezende T J R, Henry P G, Faber J, Harding I H, Ying S H, Timmann D, Thieme A, Sestakauskas K, Sarro L, Santana M M, Rylski M, Reetz K, Ratai E M, Pandolfo M, O Keefe J A, Nobre R J, Nigri A, ... Accolla E A (2026). Mri End-Points for Clinical Trials in Ataxias: Recommendations From the Ataxia Global Initiative Mri Biomarkers Working Group. Nature Reviews Neurology, 22(7), 439-456.
Chicago	Oz G, Cocozza S, Rezende T J R, Henry P G, Faber J, Harding I H, Ying S H, et al.. "Mri End-Points for Clinical Trials in Ataxias: Recommendations From the Ataxia Global Initiative Mri Biomarkers Working Group." Nature Reviews Neurology 22, no. 7 (2026): 439-456.
Harvard	Oz G et al. (2026) 'Mri End-Points for Clinical Trials in Ataxias: Recommendations From the Ataxia Global Initiative Mri Biomarkers Working Group', Nature Reviews Neurology, 22(7), pp. 439-456.
Vancouver	Oz G, Cocozza S, Rezende T J R, Henry P G, Faber J, Harding I H, et al.. Mri End-Points for Clinical Trials in Ataxias: Recommendations From the Ataxia Global Initiative Mri Biomarkers Working Group. Nature Reviews Neurology. 2026;22(7):439-456.
BibTex	@article{ author = {Oz G and Cocozza S and Rezende T J R and Henry P G and Faber J and Harding I H and Ying S H and Timmann D and Thieme A and Sestakauskas K and Sarro L and Santana M M and Rylski M and Reetz K and Ratai E M and Pandolfo M and O Keefe J A and Nobre R J and Nigri A and Nachbauer W and Rangel D M and Mochel F and Minnerop M and Marvel C L and Mariotti C and Makowska L and Lenglet C and Kovacs N and Kinnunen K M and Kapteijns K and Joers J M and Indelicato E and Heidari M and Grisoli M and Gazdzinski S and Fuin N and Franca M C JR and Ferreira M and D Ostilio K and Deistung A and Cossa A and Boesch S and Bando K and Alhusaini S and Adarmes Gomez A and Accolla E A}, title = {Mri End-Points for Clinical Trials in Ataxias: Recommendations From the Ataxia Global Initiative Mri Biomarkers Working Group}, journal = {Nature Reviews Neurology}, volume = {22}, number = {7}, pages = {439-456}, year = {2026} }
RIS	TY - JOUR AU - Oz G AU - Cocozza S AU - Rezende T J R AU - Henry P G AU - Faber J AU - Harding I H AU - Ying S H AU - Timmann D AU - Thieme A AU - Sestakauskas K AU - Sarro L AU - Santana M M AU - Rylski M AU - Reetz K AU - Ratai E M AU - Pandolfo M AU - O Keefe J A AU - Nobre R J AU - Nigri A AU - Nachbauer W AU - Rangel D M AU - Mochel F AU - Minnerop M AU - Marvel C L AU - Mariotti C AU - Makowska L AU - Lenglet C AU - Kovacs N AU - Kinnunen K M AU - Kapteijns K AU - Joers J M AU - Indelicato E AU - Heidari M AU - Grisoli M AU - Gazdzinski S AU - Fuin N AU - Franca M C JR AU - Ferreira M AU - D Ostilio K AU - Deistung A AU - Cossa A AU - Boesch S AU - Bando K AU - Alhusaini S AU - Adarmes Gomez A AU - Accolla E A TI - Mri End-Points for Clinical Trials in Ataxias: Recommendations From the Ataxia Global Initiative Mri Biomarkers Working Group JO - Nature Reviews Neurology VL - 22 IS - 7 SP - 439 EP - 456 PY - 2026 ER -

Style

Citing Format

MLA

Oz G, et al.. "Mri End-Points for Clinical Trials in Ataxias: Recommendations From the Ataxia Global Initiative Mri Biomarkers Working Group." Nature Reviews Neurology, vol. 22, no. 7, 2026, pp. 439-456.

APA

Oz G, Cocozza S, Rezende T J R, Henry P G, Faber J, Harding I H, Ying S H, Timmann D, Thieme A, Sestakauskas K, Sarro L, Santana M M, Rylski M, Reetz K, Ratai E M, Pandolfo M, O Keefe J A, Nobre R J, Nigri A, ... Accolla E A (2026). Mri End-Points for Clinical Trials in Ataxias: Recommendations From the Ataxia Global Initiative Mri Biomarkers Working Group. Nature Reviews Neurology, 22(7), 439-456.

Chicago

Oz G, Cocozza S, Rezende T J R, Henry P G, Faber J, Harding I H, Ying S H, et al.. "Mri End-Points for Clinical Trials in Ataxias: Recommendations From the Ataxia Global Initiative Mri Biomarkers Working Group." Nature Reviews Neurology 22, no. 7 (2026): 439-456.

Harvard

Oz G et al. (2026) 'Mri End-Points for Clinical Trials in Ataxias: Recommendations From the Ataxia Global Initiative Mri Biomarkers Working Group', Nature Reviews Neurology, 22(7), pp. 439-456.

Vancouver

Oz G, Cocozza S, Rezende T J R, Henry P G, Faber J, Harding I H, et al.. Mri End-Points for Clinical Trials in Ataxias: Recommendations From the Ataxia Global Initiative Mri Biomarkers Working Group. Nature Reviews Neurology. 2026;22(7):439-456.

BibTex

@article{ author = {Oz G and Cocozza S and Rezende T J R and Henry P G and Faber J and Harding I H and Ying S H and Timmann D and Thieme A and Sestakauskas K and Sarro L and Santana M M and Rylski M and Reetz K and Ratai E M and Pandolfo M and O Keefe J A and Nobre R J and Nigri A and Nachbauer W and Rangel D M and Mochel F and Minnerop M and Marvel C L and Mariotti C and Makowska L and Lenglet C and Kovacs N and Kinnunen K M and Kapteijns K and Joers J M and Indelicato E and Heidari M and Grisoli M and Gazdzinski S and Fuin N and Franca M C JR and Ferreira M and D Ostilio K and Deistung A and Cossa A and Boesch S and Bando K and Alhusaini S and Adarmes Gomez A and Accolla E A}, title = {Mri End-Points for Clinical Trials in Ataxias: Recommendations From the Ataxia Global Initiative Mri Biomarkers Working Group}, journal = {Nature Reviews Neurology}, volume = {22}, number = {7}, pages = {439-456}, year = {2026} }

RIS

TY - JOUR
AU - Oz G
AU - Cocozza S
AU - Rezende T J R
AU - Henry P G
AU - Faber J
AU - Harding I H
AU - Ying S H
AU - Timmann D
AU - Thieme A
AU - Sestakauskas K
AU - Sarro L
AU - Santana M M
AU - Rylski M
AU - Reetz K
AU - Ratai E M
AU - Pandolfo M
AU - O Keefe J A
AU - Nobre R J
AU - Nigri A
AU - Nachbauer W
AU - Rangel D M
AU - Mochel F
AU - Minnerop M
AU - Marvel C L
AU - Mariotti C
AU - Makowska L
AU - Lenglet C
AU - Kovacs N
AU - Kinnunen K M
AU - Kapteijns K
AU - Joers J M
AU - Indelicato E
AU - Heidari M
AU - Grisoli M
AU - Gazdzinski S
AU - Fuin N
AU - Franca M C JR
AU - Ferreira M
AU - D Ostilio K
AU - Deistung A
AU - Cossa A
AU - Boesch S
AU - Bando K
AU - Alhusaini S
AU - Adarmes Gomez A
AU - Accolla E A
TI - Mri End-Points for Clinical Trials in Ataxias: Recommendations From the Ataxia Global Initiative Mri Biomarkers Working Group
JO - Nature Reviews Neurology
VL - 22
IS - 7
SP - 439
EP - 456
PY - 2026
ER -

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Abstract