Life-Saving Treatments for Spinal Muscular Atrophy: Global Access and Availability.
Victor D ArmengolBasil T DarrasAhmad Abdulaziz AbulabanAli AlshehriNina BarisicTawfeg Ben-OmranGuenther BernertClaudia CastiglioniYin-Hsiu ChienMichelle Anne FarrarGwendoline Q KandawasvikaSatish V KhadilkarJean K MahChiara Marini-BettoloDamjan OsredkarGerald PfefferFlavia B PiazzonInmaculada Pitarch CastellanoSusana Quijano-RoyKayoko SaitoJin-Hong ShinJuan Francisco Vázquez CostaMaggie C WalterJithangi WanigasingheHui XiongRobert C GriggsBhaskar RoyPublished in: Neurology. Clinical practice (2023)
Overall, this survey highlights the global inequality in managing patients with SMA. The spread of newborn screening is essential in ensuring improved access to care for patients with SMA. With the advancement of neurotherapeutics, more genetic diseases will soon be treatable, and addressing the global inequality in clinical care will require novel approaches to mitigate such inequality in the future.