Nusinersen Treatment for Spinal Muscular Atrophy: Retrospective Multicenter Study of Pediatric and Adult Patients in Kuwait

• Principais autores: Asma AlTawari (Autor), Mohammad Zakaria (Autor), Walaa Kamel (Autor), Nayera Shaalan (Autor), Gamal Ahmed Ismail Elghazawi (Autor), Mohamed Esmat Anwar Ali (Autor), Dalia Salota (Autor), Amr Attia (Autor), Ehab Elsayed Ali Elanay (Autor), Osama Shalaby (Autor), Fatema Alqallaf (Autor), Vesna Mitic (Autor), Laila Bastaki (Autor)
• Formato: Livro
• Publicado em: MDPI AG, 2024-06-01T00:00:00Z.
• Assuntos: article
• Acesso em linha: Connect to this object online.

Spinal muscular atrophy is a neuromuscular genetic condition associated with progressive muscle weakness and atrophy. Nusinersen is an antisense oligonucleotide therapy approved for the treatment of 5q spinal muscular atrophy in pediatric and adult patients. The objective of this clinical case serie...