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A Prospective Observational Patient Registry to Evaluate Disease Progression in Patients With ENPP1 Deficiency and Infantile-Onset ABCC6 Deficiency (GACI Type 2)

The purpose of this prospective registry is to characterize the natural history of ectonucleotide pyrophosphatase/phosphodiesterase1(ENPP1) Deficiency and the …

Ectonucleotide Pyrophosphatase/Phosphodiesterase 1 DeficiencyATP-Binding Cassette Subfamily C Member 6 Deficiency

Inozyme PharmaNCT06302439