Novartis firm AveXis gets EC nod for Zolgensma to treat spinal muscular atrophy
The approval allows Zolgensma to treat patients with 5q SMA with a bi-allelic mutation in the SMN1 gene and a clinical diagnosis of SMA Type 1or patients with
The breakthrough therapy status will allow using of mobocertinib by patients with epidermal growth factor receptor (EGFR) exon 20 insertion mutations whose disease has advanced on or after