Ataluren improves hematopoietic and pancreatic disorders in Shwachman-Diamond syndrome patients: a compassionate program case-series

Valentino Bezzerri; Anna Pegoraro; Anca Manuela Hristodor; Genevieve M. Crane; Ilaria Meneghelli; Cecilia Brignole; Christian Boni; Elena Baldisseri; Antonio Vella; Giacomo Menichetti; Roberto Valli; Giovanni Porta; Giovanna D’Amico; Cristina Tecchio; Alice Parisi; Giuseppe Lippi; Angela Mercuri; Simone Cesaro; Seth J. Corey; Marco Cipolli

doi:10.1038/s41467-025-63137-3

Ataluren improves hematopoietic and pancreatic disorders in Shwachman-Diamond syndrome patients: a compassionate program case-series

V Valentino Bezzerri A Anna Pegoraro A Anca Manuela Hristodor G Genevieve M. Crane I Ilaria Meneghelli C Cecilia Brignole C Christian Boni E Elena Baldisseri A Antonio Vella G Giacomo Menichetti R Roberto Valli G Giovanni Porta G Giovanna D’Amico C Cristina Tecchio A Alice Parisi G Giuseppe Lippi A Angela Mercuri S Simone Cesaro S Seth J. Corey M Marco Cipolli

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Article Details

Journal Nature Communications

Volume / Issue Vol. 16, Issue 1

Published September 02, 2025

DOI 10.1038/s41467-025-63137-3

ISSN 2041-1723

Publisher Nature Portfolio

Journal Info

Nature Communications

Nature Portfolio

ISSN: 2041-1723 Open Access Life Sciences

Authors (20)

V

Valentino Bezzerri

A

Anna Pegoraro

A

Anca Manuela Hristodor

G

Genevieve M. Crane

I

Ilaria Meneghelli

C

Cecilia Brignole

C

Christian Boni

E

Elena Baldisseri

A

Antonio Vella

G

Giacomo Menichetti

R

Roberto Valli

G

Giovanni Porta

G

Giovanna D’Amico

C

Cristina Tecchio

A

Alice Parisi

G

Giuseppe Lippi

A

Angela Mercuri

S

Simone Cesaro

S

Seth J. Corey

M

Marco Cipolli

Ataluren improves hematopoietic and pancreatic disorders in Shwachman-Diamond syndrome patients: a compassionate program case-series

Article Details

Journal Info

Authors (20)

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