3 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*…
To evaluate the ability of ataluren to improve pulmonary function relative to placeboTo determine the effect of ataluren on:1. Pulmonary symptoms2. General well-being3. Health-related Quality Life (HRQL)
Main objective:To explore the impact of setmelanotide on obesity in patients with various specific rare genetic mutations.Secondary objective:To assess the effects of setmelanotide on:* Safety and tolerability-Hunger* Waist circumferenceOptional sub…