Phase I clinical evaluation of CNSA-001 (sepiapterin), a novel pharmacological treatment for phenylketonuria and tetrahydrobiopterin deficiencies, in healthy volunteers.

Smith, Neil

Phase I clinical evaluation of CNSA-001 (sepiapterin), a novel pharmacological treatment for phenylketonuria and tetrahydrobiopterin deficiencies, in healthy volunteers. [electronic resource] - Molecular genetics and metabolism 04 2019 - 406-412 p. digital

Publication Type: Clinical Trial, Phase I; Journal Article; Randomized Controlled Trial

1096-7206

10.1016/j.ymgme.2019.02.001 doi


Administration, Oral
Adult
Australia
Biopterins--analogs & derivatives
Dose-Response Relationship, Drug
Double-Blind Method
Drug Compounding
Female
Healthy Volunteers
Humans
Male
Phenylalanine
Phenylketonurias--drug therapy
Pterins--administration & dosage
Serotonin