Patisiran in ATTRv amyloidosis with polyneuropathy: “PatisiranItaly” multicenter observational study
Patisiran can be considered a valid therapeutic option for the management of patients with ATTRv amyloidosis. Considering its mechanism of action, similar outcomes could also be expected with the wider utilization of newly approved gene silencers for ATTRv therapy, such as vutrisiran.
Authors
- Stefano Tozza (ORCID: https://orcid.org/0000-0002-9672-4577)
- Giammarco Milella (ORCID: https://orcid.org/0000-0002-7612-382X)
- Simone Longhi (ORCID: https://orcid.org/0000-0002-7512-1179)
- Marco Currò Dossi
- Luca Gentile (ORCID: https://orcid.org/0000-0001-7947-8610)
- Massimo Russo (ORCID: https://orcid.org/0000-0002-6017-370X)
- A Lotti (ORCID: https://orcid.org/0000-0001-8121-2627)
- M Burattini (ORCID: https://orcid.org/0009-0007-9656-5311)
- Francesca Vitali (ORCID: https://orcid.org/0000-0002-3177-1575)
- Luigi Sellitti
- Loris Poli (ORCID: https://orcid.org/0000-0003-4912-818X)
- Marco Luigetti (ORCID: https://orcid.org/0000-0001-7539-505X)
- Massimiliano Filosto (ORCID: https://orcid.org/0000-0002-2852-7512)
- Ilaria Cani (ORCID: https://orcid.org/0000-0002-9189-1982)
- Lorenzo Verriello (ORCID: https://orcid.org/0000-0002-5301-8621)
- Chiara Briani (ORCID: https://orcid.org/0000-0001-8035-0200)
- Sabrina Gasverde
- Pietro Guaraldi (ORCID: https://orcid.org/0000-0003-0597-603X)
- Ângela Romano (ORCID: https://orcid.org/0000-0002-2061-1509)
- Alessandro Barilaro (ORCID: https://orcid.org/0000-0001-7418-7152)
- Vittoria Cianci (ORCID: https://orcid.org/0000-0002-2352-5710)
- Filippo Brighina (ORCID: https://orcid.org/0000-0001-5875-3126)
- Giovanni Palumbo (ORCID: https://orcid.org/0000-0003-3757-4967)
- Daniela Di Lisi (ORCID: https://orcid.org/0000-0003-4510-6805)
- Luca Pradotto (ORCID: https://orcid.org/0000-0003-1103-1964)
- Matteo Tagliapietra (ORCID: https://orcid.org/0000-0002-3048-1453)
- Luca Leonardi (ORCID: https://orcid.org/0000-0002-1267-864X)
- Yuri Falzone (ORCID: https://orcid.org/0000-0003-3233-1907)
- Alessandro Salvalaggio (ORCID: https://orcid.org/0000-0002-1273-7566)
- Chiara Gemelli (ORCID: https://orcid.org/0000-0003-1911-2726)
- Giovanni Antonini (ORCID: https://orcid.org/0000-0002-7519-7739)
- Anna Mazzeo (ORCID: https://orcid.org/0000-0003-1914-6184)
- Vincenzo Di Stefano (ORCID: https://orcid.org/0000-0001-9805-1655)
- Mara Turri (ORCID: https://orcid.org/0000-0001-9130-1295)
- Marco Ceccanti (ORCID: https://orcid.org/0000-0001-8897-3736)
- Giulia Carlini (ORCID: https://orcid.org/0000-0001-9061-2462)
- Antonio Di Muzio (ORCID: https://orcid.org/0000-0003-0471-0192)
- Mariangela Goglia (ORCID: https://orcid.org/0009-0006-4128-5876)
- Pietro Cortelli (ORCID: https://orcid.org/0000-0002-3633-8818)
- Maria Ausilia Sciarrone (ORCID: https://orcid.org/0000-0002-1490-0320)
- Fiore Manganelli
- Giuseppina Novo
- Cristina Petrelli
- Silvia Fenu
Institutions
- Università Cattolica del Sacro Cuore (IT)
- University of Messina (IT)
- University of Rome Tor Vergata (IT)
- University of Verona (IT)
- Marche Polytechnic University (IT)
- University of Padua (IT)
- Agostino Gemelli University Polyclinic (IT)
- San Raffaele University of Rome (IT)
- University of Chieti-Pescara (IT)
- Ospedale di Bolzano (IT)
- Saint Camillus International University of Health and Medical Sciences (IT)
- Azienda ospedaliera "Bianchi-Melacrino-Morelli" (IT)
- Azienda Socio Sanitaria Territoriale degli Spedali Civili di Brescia (IT)
- CTO Andrea Alesini (IT)
- Azienda USL di Bologna (IT)
- Istituto delle Scienze Neurologiche di Bologna (IT)
- Ospedale Santa Maria della Misericordia di Udine (IT)
- Ospedale Infermi di Rimini (IT)
- IRCCS Istituto Auxologico Italiano (IT)
- Ospedale Policlinico San Martino (IT)
- Azienda Ospedaliera Universitaria Policlinico "Paolo Giaccone" di Palermo (IT)
- Istituti di Ricovero e Cura a Carattere Scientifico (IT)
- Fondazione IRCCS Istituto Neurologico Carlo Besta (IT)
- University of Florence (IT)
- University of Macerata (IT)
- University of Turin (IT)
- University of Bari Aldo Moro (IT)
- University of Naples Federico II (IT)
- University of Brescia (IT)
- Sapienza University of Rome (IT)
- University of Palermo (IT)
Publication Details
- Journal
- Journal of Neurology
- Published
- 2025-02-15
- DOI
- https://doi.org/10.1007/s00415-025-12950-3
- Citations
- 9
- Primary Topic
- Amyloidosis: Diagnosis, Treatment, Outcomes
- Type
- article
- Field-Weighted Citation Impact
- 7.25
Funders
- Università Cattolica del Sacro Cuore