Proximal spinal muscular atrophy (SMA) is an inherited neurodegenerative disease with a heterogeneous clinical phenotype. Although there is no cure for SMA, several strategies are currently being developed. In this review, we summarize the ongoing clinical trials and molecular mechanisms of successful approaches to SMA treatment.
Spinal muscular atrophy, therapeutic approaches, clinical trials
BORA, GAMZE; YEŞBEK-KAYMAZ, AYŞE; BEKİRCAN-KURT, CAN EBRU; HALİLOĞLU, VİLDAN GÖKNUR; TOPALOĞLU, HALUK AYDIN; ERDEM-YURTER, HAYAT; and ERDEM-ÖZDAMAR, SEVİM
"Recent therapeutic developments in spinal muscular atrophy,"
Turkish Journal of Medical Sciences: Vol. 48:
2, Article 1.
Available at: https://journals.tubitak.gov.tr/medical/vol48/iss2/1