The US Food and Drug Administration (FDA) has granted rare paediatric disease designation to Vanda Pharmaceuticals’ VCA-894A, an investigational antisense oligonucleotide therapy for Charcot-Marie-Tooth disease, axonal, type 2S (CMT2S).
Through a multi-phase collaboration, WuXi will leverage and evaluate Cyclica’s cloud-based proteome Ligand Express screening platform to investigate the off-target profiles of small molecules, apply Cyclica’s novel and
At the week 16 primary efficacy analysis, the study met both co-primary endpoints and all key secondary endpoints. Fasinumab-treated patients experienced significantly less pain and significantly improved functional
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