Bioequivalence Study of Two Oral Nitisinone Formulations to Treat Hereditary Tyrosinemia (HT-1)

计划入组 24 人 · 首次发布于 2016 年 4 月 25 日

Bioavailability Food-Effect Study of an Oral Nitisinone Formulation to Treat Hereditary Tyrosinemia (HT-1)

计划入组 20 人 · 首次发布于 2016 年 4 月 25 日

Hereditary Hepatorenal Tyrosinemia Natural History in Egypt and the Arab World (Multicenter Clinical Study)

计划入组 50 人 · 首次发布于 2018 年 2 月 27 日

Nitisinone(Nityr, Orfadin)

C₁₄H₁₀F₃NO₅ · Rx

RNA actuator therapeutic for hereditary angioedema(CAMP4 Therapeutics)

RNA actuator therapeutic (hereditary angioedema)

Biora Therapeutics

United States

Astria Therapeutics Inc

United States

Pharming Group

Netherlands

BioCryst Pharmaceuticals Divests European ORLADEYO Rights for $264 Million to Achieve Debt-Free Status

Faeth Therapeutics Raises $25M to Advance PIKTOR Therapy After 80% Response Rate in Endometrial Cancer

FDA Accepts Donidalorsen NDA for Hereditary Angioedema Prophylaxis