A contemporary analysis of the Australian clinical and genetic landscape of spinal muscular atrophy: a registry based studyResearch in context

• Main Authors: Lakshmi Balaji (Author), Robin Forbes (Author), Anita Cairns (Author), Hugo Sampaio (Author), Andrew J. Kornberg (Author), Lauren Sanders (Author), Phillipa Lamont (Author), Christina Liang (Author), Kristi J. Jones (Author), Kristen Nowak (Author), Cullen O'Gorman (Author), Ian Woodcock (Author), Nancy Briggs (Author), Eppie M. Yiu (Author), Michelle A. Farrar (Author), Didu Kariyawasam (Author)
• Format: Book
• Published: Elsevier, 2024-12-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Summary: Background: New paradigms of diagnosis and treatment have changed the neurodegenerative trajectory for individuals with spinal muscular atrophy (SMA). Registries are a critical tool to provide real-world data on treatment patterns, their effects and health care provision within this evolvin...