Lucerastat, an oral therapy for Fabry disease: results from a pivotal randomized phase 3 study and its open-label extension

Peter Nordbeck; Ozlem Goker-Alpan; John A. Bernat; Dominique P. Germain; Pilar Giraldo; Ana Jovanović; Virginia Kimonis; Kathleen Nicholls; Cheryl Rockman-Greenberg; Raphael Schiffmann; Mark Thomas; Anna Tylki-Szymanska; Eric Wallace; Richard W. D. Welford; Michael L. West; Martine Clozel; Aline Frey; Luba Trokan; Markus S. Mueller; Markus Vogler; Christoph Wanner; Derralynn Hughes

doi:10.1038/s41467-025-68256-5

Lucerastat, an oral therapy for Fabry disease: results from a pivotal randomized phase 3 study and its open-label extension

P Peter Nordbeck O Ozlem Goker-Alpan J John A. Bernat D Dominique P. Germain P Pilar Giraldo A Ana Jovanović V Virginia Kimonis K Kathleen Nicholls C Cheryl Rockman-Greenberg R Raphael Schiffmann M Mark Thomas A Anna Tylki-Szymanska E Eric Wallace R Richard W. D. Welford M Michael L. West M Martine Clozel A Aline Frey L Luba Trokan M Markus S. Mueller M Markus Vogler C Christoph Wanner D Derralynn Hughes

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Article Details

Journal Nature Communications

Volume / Issue Vol. 17, Issue 1

Published January 10, 2026

DOI 10.1038/s41467-025-68256-5

ISSN 2041-1723

Publisher Nature Portfolio

Journal Info

Nature Communications

Nature Portfolio

ISSN: 2041-1723 Open Access Life Sciences

Authors (22)

P

Peter Nordbeck

O

Ozlem Goker-Alpan

J

John A. Bernat

D

Dominique P. Germain

P

Pilar Giraldo

A

Ana Jovanović

V

Virginia Kimonis

K

Kathleen Nicholls

C

Cheryl Rockman-Greenberg

R

Raphael Schiffmann

M

Mark Thomas

A

Anna Tylki-Szymanska

E

Eric Wallace

R

Richard W. D. Welford

M

Michael L. West

M

Martine Clozel

A

Aline Frey

L

Luba Trokan

M

Markus S. Mueller

M

Markus Vogler

C

Christoph Wanner

D

Derralynn Hughes

Lucerastat, an oral therapy for Fabry disease: results from a pivotal randomized phase 3 study and its open-label extension

Article Details

Journal Info

Authors (22)

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