Rare diseases
Investigational molecules
Locations
Phase 2 Study of AGA2115 in Adults with Osteogenesis Imperfecta (COL1A1/COL1A2 Variants) Evaluating Bone Density
The NetherlandsLong‑term Safety and Tolerability of Admilparant in Adults with Idiopathic or Progressive Pulmonary Fibrosis
The NetherlandsEvaluation of recurrence‑free survival with cemiplimab immunotherapy in medically inoperable stage I non‑small cell lung cancer patients
The NetherlandsLong-Term Safety and Efficacy of Oral Ribitol in Participants with Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I/R9)
The NetherlandsSafety and Efficacy of TSRA-196 (rnaivt9315 and rnacs24757) in Adults with Lung or Liver Disease due to Alpha‑1 Antitrypsin Deficiency
The NetherlandsSafety and pharmacodynamics of cangrelor tetrasodium in paediatric patients with congenital heart disease undergoing percutaneous vascular procedures
The NetherlandsPhase 3 Randomized Double‑Blind Placebo‑Controlled Study of Intravenous sgt-003 in Ambulatory Male Patients with Duchenne Muscular Dystrophy
The NetherlandsPhase II/III trial of autologous dendritic cells loaded with allogenic mesothelioma lysate in patients with borderline resectable pancreatic cancer
The NetherlandsA study to evaluate the safety and effectiveness of petosemtamab and pembrolizumab in adults with non-small cell lung cancer
The NetherlandsA study of rilvegostomig and ramucirumab in patients with advanced or metastatic non-small cell lung cancer
The NetherlandsA study to evaluate the effectiveness and safety of admilparant in patients with progressive pulmonary fibrosis
The NetherlandsPhase 3 Randomized Study of Niraparib Tosilate Monohydrate vs. Temozolomide in Newly Diagnosed MGMT Unmethylated Glioblastoma
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