source
- PepGen reports positive Phase 2 data for PGN-EDODM1 in myotonic dystrophyPepGen disclosed that its experimental oligonucleotide therapy PGN-EDODM1 showed encouraging early signals in a Phase 2 cohort of patients with myotonic dystrophy type 1 (DM1). The company said the…
- FDA opens investigation into Novartis' Zolgensma after cancer cases reportedThe U.S. Food and Drug Administration announced on Friday that it is opening a formal investigation into the gene-therapy product Zolgensma, marketed by Novartis for spinal muscular atrophy. The a…