Current Trials

Syntis Bio: Enzyme Substitution Therapy

Tome Bio: Gene Therapy

Travere Therapeutics: Enzyme Replacement Therapy

Natural History Study of CBS Deficiency

For the most current,  up-to-date list of clinical trials and studies involving HCU, CBS, MTHFR and Cbl, please visit the Clinical Trials website

Discontinued programs

Aeglea BioTherapeutics: Enzyme Replacement Therapy

Synlogic: Probiotic Enzyme

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