Spinal muscular atrophy with respiratory distress type 1

Could an outcome-based agreement be operationalized using real-world data from the Canadian Neuromuscular Disease Registry? Perspectives from an expert-led assessment in spinal muscular atrophy

Published in Ther Adv Rare Dis. Mitha A et al.

The post-pandemic pivot: mRNA therapeutics enter the chronic rare disease arena

Published in Mol Ther Nucleic Acids. Giangrande PH et al.

The impact of antisense oligonucleotide (ASO) therapeutics on the future of rare disease drug discovery

Published in Expert Opin Drug Discov. Ruchi R et al.

EMA Grants Conditional Approval for Novel SMA Therapy

EMA granted conditional authorization to a new oral therapy for spinal muscular atrophy.