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
2025-09-02