Rare diseases
Investigational molecules
Locations
Efficacy and Safety of Intranasal Cenegermin in Adults with Non‑Arteritic Anterior Ischemic Optic Neuropathy (NAION)
PortugalEpcoritamab plus rituximab versus rituximab alone in elderly, frail or unfit patients with newly diagnosed diffuse large B‑cell lymphoma
PortugalSafety and Efficacy of Intrathecal BIIB115 in Infants with Spinal Muscular Atrophy Previously Treated with Onasemnogene Abeparvovec
PortugalPhase 1/2 Study of ter-2013-01, imlunestrant, and fulvestrant in patients with solid tumors with AKT/PI3K/PTEN pathway alterations
PortugalStudy of Crinecerfont in Adults with Classic Congenital Adrenal Hyperplasia to Reduce Androgen Levels
PortugalEfficacy and Safety of Pegcetacoplan in Adults and Adolescents with Focal Segmental Glomerulosclerosis: Phase 2/3 Open‑Label and Randomized Placebo‑Controlled Study
PortugalPhase 2/3 study of telisotuzumab adizutecan with drug combination versus standard care in adults with first‑line metastatic pancreatic ductal adenocarcinoma
PortugalSafety and Tolerability of Intrathecal ION337 in Patients with Dravet Syndrome
PortugalLong‑Term Safety and Tolerability of Treprostinil Palmitil Inhalation Powder in Patients with Pulmonary Hypertension Associated with Interstitial Lung Disease
PortugalLong‑term Safety and Tolerability of Admilparant in Adults with Idiopathic or Progressive Pulmonary Fibrosis
PortugalSafety and pharmacodynamics of cangrelor tetrasodium in paediatric patients with congenital heart disease undergoing percutaneous vascular procedures
PortugalSafety and Efficacy of VX-828, Deutivacaftor, and Tezacaftor in Adults with Cystic Fibrosis
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