In this issue:
ENVISION: givosiran ‘interferes’ with acute intermittent porphyria
FIREFISH: risdiplam demonstrates significant improvements in motor function for SMA
Could doublet or triplet regimens of gene therapies be beneficial for SMA?
Long-term data from START finds onasemnogene abeparvovec safe and efficacious for SMA
Nusinersen extends survival and improves motor function in infants with SMA
Long-term safety and efficacy of lumacaftor/ivacaftor in children with F508del-CFTR-mutated cystic fibrosis Tezacaftor/ivacaftor safe and efficacious for CFTR-mutated cystic fibrosis
Triplet elexacaftor/tezacaftor/ ivacaftor efficacious in children with F508del-CFTR-mutated cystic fibrosis Elexacaftor/tezacaftor/ivacaftor use in France leads to reductions in lung transplantation
First-in-human trial of oral omaveloxolone for advanced solid tumours
Please login below to download this issue (PDF)