Flow diagram of Study 250-201.
In Part 1, 3 patients, i.e., 9001, 9002, and 9003, were recruited and treated with escalating doses of 30, 100, and 300 mg tralesinidase alfa as described in Results. These 3 patients were eventually recruited to Part 2 of the study and treated for an additional 48 weeks. In addition, 19 patients, i.e., patients 9004–9015 and 9017–9023, previously observed in our natural history Study 250-901 (