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A Multicenter, Randomized, Double blind, Vehicle-controlled, Phase 3 Efficacy and Safety Study of Patidegib Topical Gel, 2%, for the Reduction of Disease Burden of Persistently Developing Basal Cell Carcinomas (BCCs) in Subjects with Basal Cell Nevus Syndrome

Primary Objective:To assess the number of new BCCs in the 2 arms (Patidegib Topical Gel, 2%, and Vehicle (placebo)) when applied twice daily to the face of subjects with Gorlin Syndrome.Secondary Objective:To assess the safety and tolerability of…

Treatment of Skin Severity in Ichthyosis with Hyperbaric Oxygen Therapy

To assess clinical improvement of skin severity in patients with ichthyosis

Transvamix (10% THC / 5% CBD) to treat chronic pain in Epidermolysis Bullosa: An explorative randomized, placebo-controlled and double-blind intervention crossover study

The primary objective is to determine the effect of Transvamix on the pain quality item *unpleasantness*, relative to placebo. Secondary objectives include the assessment of general pain qualities, pain self-efficacy, general pain and pruritus…

A Phase II, double-blind, placebo-controlled, randomized, cross-over, dose-ranging study of oral PHA-022121 for acute treatment of angioedema attacks in patients with hereditary angioedema due to C1-inhibitor deficiency type I and II

The purpose of this study is to find out how safe and effective the new drug PHA-022121 is for the treatment of Hereditary Angioedema. The main purpose of this research study is to test the effectiveness of 3 different doses of PHA-022121 in…