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Biopharma · global

AMO-02 Trial Design Gains Regulatory Alignment Across Three Regions, Bringing a Clearer Path for cDM1 Pediatric Drug Development

For a rare and complex childhood disease such as congenital myotonic dystrophy, regulators’ agreement on how a trial should be conducted is often as critical as the drug itself. AMO Pharma’s latest progress suggests that AMO-02 is seeking to turn uncertain clinical development questions into an executable registration study design.