Shots:
CSL has reported topline P-IIIb results evaluating Andembry (garadacimab-gxii) in children aged 2 to 11yrs. with hereditary angioedema (HAE)
Pts aged 6–11 years received 100mg (QM, n=16), while those aged 2–5yrs. received 100mg (Q2M, n=6). Andembry showed a favorable safety profile & treatment response, with most pts remaining attack-free over 12mos.
Full results will be…
Shots:
The EC has approved label extension of Evrysdi (5mg oral tablet) for pts with spinal muscular atrophy (SMA)
Approval was based on P-I study assessing Evrysdi 5mg tablet vs oral solution, showing bioequivalence with comparable efficacy & safety; data was presented at SMA Europe 2024
Evrysdi, an SMN2 splicing modifier, treats SMA caused by…

