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- Novartis' Zolgensma linked to tumors as FDA probes gene therapy and orders GLP-1 eye risk warningsNovartis confirmed that nine patients who received its Zolgensma gene therapy for spinal muscular atrophy have developed tumors, prompting the FDA to open an investigation into the approved treatme…
- PepGen stock surges as DM1 oligonucleotide PGN-EDODM1 shows record splicing correction in Phase 2PepGen reported that its experimental oligonucleotide PGN-EDODM1 achieved a mean splicing correction of 46.5% at week 13 in a Phase 2 trial for myotonic dystrophy type 1 (DM1). This performance exc…