Treatment of spinal muscular atrophy with Onasemnogene Abeparvovec in Switzerland: a prospective observational case series study

Abstract Background Spinal muscular atrophy (SMA) is a rare neuromuscular disorder leading to early death in the majority of affected individuals without treatment. Recently, targeted treatment approaches including Onasemnogene Abeparvovec (OA) were introduced. This study describes the first real-wo...

Deskribapen osoa

Xehetasun bibliografikoak

Egile Nagusiak:	Georg M. Stettner, Oswald Hasselmann, Anne Tscherter, Elea Galiart, David Jacquier, Andrea Klein
Formatua:	Artigo
Hizkuntza:	Inglês
Argitaratua:	BMC 2023-02-01
Saila:	BMC Neurology
Gaiak:	Spinal muscular atrophy Onasemnogene Abeparvovec Zolgensma Gene therapy Switzerland
Sarrera elektronikoa:	https://doi.org/10.1186/s12883-023-03133-6
Etiketak:	Etiketa erantsi Etiketarik gabe, Izan zaitez lehena erregistro honi etiketa jartzen!