Olipudase alfa for acid sphingomyelinase deficiency: Evidence of efficacy from a compassionate use program

Vincenza Gragnaniello, Veronica Maria Tagi, Francesca Carubbi, Antonio Barbato, Michelina Sibilio, Simona Fecarotta, Maria Grazia Pappalardo, Irene Motta, Annalisa Sechi, Maurizio Scarpa, Andrea Pession, Laura Fiori

Open source

DOI
10.1016/j.ymgmr.2026.101372
Published
2026-12
Container
Molecular Genetics and Metabolism Reports
Publisher
Elsevier BV
Open access
unknown

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BibTeX

@article{allodium:10.1016/j.ymgmr.2026.101372,
  title = {Olipudase alfa for acid sphingomyelinase deficiency: Evidence of efficacy from a compassionate use program},
  author = {Vincenza Gragnaniello and Veronica Maria Tagi and Francesca Carubbi and Antonio Barbato and Michelina Sibilio and Simona Fecarotta and Maria Grazia Pappalardo and Irene Motta and Annalisa Sechi and Maurizio Scarpa and Andrea Pession and Laura Fiori},
  year = {2026},
  journal = {Molecular Genetics and Metabolism Reports},
  doi = {10.1016/j.ymgmr.2026.101372},
  url = {https://doi.org/10.1016/j.ymgmr.2026.101372}
}

RIS

TY  - JOUR
TI  - Olipudase alfa for acid sphingomyelinase deficiency: Evidence of efficacy from a compassionate use program
AU  - Vincenza Gragnaniello
AU  - Veronica Maria Tagi
AU  - Francesca Carubbi
AU  - Antonio Barbato
AU  - Michelina Sibilio
AU  - Simona Fecarotta
AU  - Maria Grazia Pappalardo
AU  - Irene Motta
AU  - Annalisa Sechi
AU  - Maurizio Scarpa
AU  - Andrea Pession
AU  - Laura Fiori
PY  - 2026
JO  - Molecular Genetics and Metabolism Reports
DO  - 10.1016/j.ymgmr.2026.101372
UR  - https://doi.org/10.1016/j.ymgmr.2026.101372
ER  - 

APA

Gragnaniello, V., Tagi, V. M., Carubbi, F., Barbato, A., Sibilio, M., Fecarotta, S., Pappalardo, M. G., Motta, I., Sechi, A., Scarpa, M., Pession, A., & Fiori, L. (2026). Olipudase alfa for acid sphingomyelinase deficiency: Evidence of efficacy from a compassionate use program. Molecular Genetics and Metabolism Reports. https://doi.org/10.1016/j.ymgmr.2026.101372

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