Rare diseases
Investigational molecules
Locations
Study of alpelisib in children and adults with PIK3CA-Related Overgrowth Spectrum (PROS)
FranceStudy of acellular pertussis vaccine and its effect on whooping cough colonization in healthy adults using controlled human infection
The NetherlandsStudy of VRDN-001 compared to placebo to evaluate safety and effectiveness in adults with chronic thyroid eye disease
FranceStudy of BMN 351 given by intravenous infusion to evaluate safety and tolerability in patients with Duchenne Muscular Dystrophy
ItalyStudy of efavaleukin alfa safety and effectiveness in adults with moderate to severe ulcerative colitis
AustriaStudy of Efavaleukin Alfa to Treat Adults with Moderate to Severe Ulcerative Colitis
AustriaStudy of rimegepant for preventing chronic migraine in adolescents aged 12 to 17 years
CzechiaStudy of Datopotamab Deruxtecan with or without Durvalumab compared to standard treatment for patients with Stage I-III triple-negative breast cancer who have remaining cancer after initial therapy
BelgiumStudy of bemarituzumab treatment for patients with solid tumors that have high levels of FGFR2b protein
AustriaStudy of SAR441566 and methotrexate in adults with moderate to severe rheumatoid arthritis
CzechiaStudy of Tozorakimab to reduce exacerbations in former smokers with chronic obstructive pulmonary disease (COPD)
FranceLong-term safety study of NNC6019-0001 for patients with transthyretin amyloid cardiomyopathy (ATTR-CM)
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