Modifying disease registries to address the evolving field in rare diseases: the iSMAc/ITASMAc experience in spinal muscular atrophy

IntroductionThe availability of disease modifying therapies in Spinal Muscular Atrophy (SMA) has highlighted the need for more structured registries for collecting reliable information both in treated and untreated individuals. Regulators recommendations have recently become available suggesting how...

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Номзүйн дэлгэрэнгүй

Үндсэн зохиолчид:	Giorgia Coratti, Chiara Bravetti, Gianpaolo Cicala, Chiara Cutrì, Valeria A. Sansone, Adele D’Amico, Claudio Bruno, Sonia Messina, Federica Ricci, Tiziana Mongini, Michela Coccia, Elena Pegoraro, Riccardo Masson, Angela Berardinelli, Caterina Agosto, Antonella Pini, Antonio Varone, Mara Turri, Massimiliano Filosto, Giacomo Comi, Lorenzo Maggi, Irene Bruno, Maria Grazia D'Angelo, Antonio Trabacca, Veria Vacchiano, Michele Sacchini, Delio Gagliardi, Eustachio D’Errico, Lucia Ruggiero, Lorenzo Verriello, Filippo Brighina, Matteo Garibaldi, Riccardo Zuccarino, Vincenzo Nigro, Roberta Battini, Giulia Ricci, Sabrina Siliquini, Alberto A. Zambon, Betty Polikar, Maria Carmela Pera, Marika Pane, Eugenio Mercuri, ITASMAc Working Group
Формат:	Artigo
Хэл сонгох:	Inglês
Хэвлэсэн:	Frontiers Media S.A. 2026-06-01
Цуврал:	Frontiers in Neurology
Нөхцлүүд:	disease registry longitudinal data neuromuscular disorders real-world data spinal muscular atrophy
Онлайн хандалт:	https://www.frontiersin.org/articles/10.3389/fneur.2026.1833889/full
Шошгууд:	Шошго нэмэх Шошго байхгүй, Энэхүү баримтыг шошголох эхний хүн болох!