2 results
Approved WMOCompleted
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)
Approved WMOPending
To describe and investigate safety and tolerability of the intradermal delivery of two fractional doses of 3/3 µg Comirnaty Original/Omicron BA.4-5 vaccin in patients with Fibrodysplasia Ossificans Progressiva. Additionally to measure the…