AZ declares grant support paid to their institution from Takeda, Sanofi, Pfizer, and Centogene; consulting fees from Takeda, Prevail Therapeutics, BioEvents, Pfizer, AVROBIO, MLC Pharma, and Insighteq; and speaking fees and travel costs from Takeda, BioEvents, and Pfizer. AZ has stock or stock options in Protalix and Agyany Pharmaceuticals. SR-V declares grant support paid to their institution from Takeda, Sanofi, Pfizer, and Centogene; consulting fees, speaking fees, and travel costs from Takeda, Pfizer, and Sanofi; and is a member of the Prevail Therapeutics advisory board. MB-C declares grant support paid to their institution from Takeda, Sanofi, Pfizer, and Centogene; and travel costs from Takeda and BioEvents. MI declares grant support paid to their institution from Takeda, Sanofi, Pfizer, and Centogene; speaking fees from Takeda and Pfizer; and travel costs paid to their institution from Takeda, BioEvents, Pfizer, and Sanofi. AR declares no competing interests.