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- Beam Therapeutics reports early Phase 1/2 data for base editing therapy in GSD Ia, partners with Pfizer and EnodiaBeam Therapeutics disclosed early results from a Phase 1/2 study evaluating a base editing approach aimed at correcting the genetic defect that causes glycogen storage disease type Ia, a rare metab…
- Patient advocates and experts raise alarm over safety of Neurocrine Biosciences rare disease drugTwo months after an open letter highlighted possible safety issues, patient advocacy groups and several experts are publicly voicing growing alarm about a rare disease therapy marketed by Neurocrin…