Vanda Pharmaceuticals Granted Green Light by FDA to Advance with Experimental Antisense Oligonucleotide Therapy, VCA-894A, for Managing Charcot-Marie-Tooth Disease, Subtype 2S.
Strand Therapeutics recently revealed that the FDA has approved its request to commence a Phase 1 clinical trial, marking the initial human study of STX-001.
Ionis reveals encouraging preliminary outcomes from the third phase of the OASIS-HAE trial, examining the experimental compound donidalorsen in individuals with genetic angioedema.