Rare diseases
Investigational molecules
Locations
Switching to lenacapavir-based drug combination vs cabotegravir-based drug combination in virologically suppressed adults with HIV-1
PolandZodasiran for Adolescents With Homozygous Familial Hypercholesterolemia: A Phase 3 Study of Efficacy and Safety
PolandEfficacy and safety of votoplam in adults with Huntington’s disease: a randomized, double‑blind, placebo‑controlled Phase 3 trial
PolandPhase 2 Randomized Double‑Blind Placebo‑Controlled Study of MH002 in Adults with Mild‑to‑Moderate Ulcerative Colitis Inadequately Controlled by 5‑ASA
PolandComparing Daraxonrasib Alone or Combined with Gemcitabine and Paclitaxel Albumin‑Bound vs Standard Chemotherapy in Patients with Metastatic Pancreatic Adenocarcinoma
PolandA Study to Assess Safety and Efficacy of Surzetoclax Alone or with Etentamig in Adults with Relapsed or Refractory Multiple Myeloma
PolandA Phase 2a Study of Empasiprubart IV Monotherapy in Adults with AChR‑Ab Seropositive Generalized Myasthenia Gravis
PolandCabozantinib Maintenance Therapy vs Best Supportive Care in Osteosarcoma Patients in Complete Remission or with Residual Disease after First‑Line Chemotherapy
PolandCevostamab, Pomalidomide and Dexamethasone in Patients with Relapsed or Refractory Multiple Myeloma Previously Treated with 1‑3 Lines of Therapy
PolandSafety, Pharmacokinetics, and Exploratory Efficacy of BI 1015550 and Nerandomilast in Children and Adolescents Aged 2‑17 Years With Fibrosing Interstitial Lung Disease
PolandPhase 3 Study Comparing JNJ-79635322 with Teclistamab in Patients with Relapsed or Refractory Multiple Myeloma after 1‑3 Prior Treatments
PolandStudy of Benralizumab Safety, Pharmacokinetics, and Efficacy in Children with Eosinophilic Disease
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