Quality of Life Assessment in Romanian Patients with Spinal Muscular Atrophy Undergoing Nusinersen Treatment

• Glavni autori: Bogdana Cavaloiu (Autor), Iulia-Elena Simina (Autor), Lazar Chisavu (Autor), Crisanda Vilciu (Autor), Iuliana-Anamaria Trăilă (Autor), Maria Puiu (Autor)
• Format: Knjiga
• Izdano: MDPI AG, 2024-08-01T00:00:00Z.
• Teme: article
• Online pristup: Connect to this object online.

Spinal muscular atrophy (SMA), identified over a century ago, is characterized by severe muscle wasting and early mortality. Despite its rarity, the high carrier frequency of the responsible genetic mutations and the variability in its manifestations make it a significant research focus. This prospe...