Adaptimmune to present T cells clinical trial results
The patients in single arm open label extension study were given autologous stem cell transplant along with modified T cells. The clinical trial, which enrolled 6 patients, aimed
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).