4 results
The primary objectives of this study are: * To characterize the effects of 150 mg and 450 mg of AT1001 administered 2 hours before administration of agalsidase on the safety and plasma pharmacokinetics of agalsidase in subjects with Fabry Disease*…
Primary Objective• To estimate the long term safety of 100 mg and 200 mg once daily (QD) of abrocitinib with or without topical treatments in adult and adolescent subjects who previously participated in qualifying abrocitinib atopic dermatitis (AD)…
Primary Objective:To evaluate the efficacy of taldefgrobep alfa in participants who are already taking a stable dose of nusinersen or risdiplam or have a history of onasemnogene abeparvovec-xioi, compared to placebo, measured by change in the 32…
Primary:To provide access to abrocitinib to adolescent and adult patients with or without background topical therapy who have inadequate treatment options due to inadequate response or intolerance to available approved medicated topical and systemic…