Rare diseases
Investigational molecules
Locations
Study on Gene Therapy AAVAnc80-hOTOF for Patients with Sensorineural Hearing Loss from Otoferlin Gene Mutations
SpainStudy of ABTL0812 with Irinotecan and Temozolomide for Children with Relapsed or Refractory Neuroblastoma and Other Solid Tumors
SpainStudy on Cefiderocol for Treating Gram-negative Bacterial Infections in Hospitalized Infants Under 3 Months
SpainStudy on the Safety and Tolerability of Sisunatovir in Infants and Children with RSV Infection
SpainStudy on Gene Therapy with AAVrh10 for Infants with Krabbe Disease Undergoing Stem Cell Transplantation
SpainStudy of rocatinlimab (AMG 451) in adolescents with moderate to severe atopic dermatitis to evaluate its effectiveness and safety over 52 weeks
SpainStudy of Ruxolitinib Cream for Children and Adolescents Ages 6-17 with Moderate Atopic Dermatitis Who Did Not Respond Well to Standard Treatments
SpainStudy on the Effects of TAK-881 and Hyaluronidase in Adults with Primary Immunodeficiency Diseases
SpainStudy on Long-term Safety of TAK-881 (Human Normal Immunoglobulin, Hyaluronidase) for Patients with Primary Immunodeficiency Diseases
SpainStudy of Baricitinib for Children and Adolescents with Moderate-to-Severe Atopic Dermatitis
SpainStudy on Long-Term Safety and Effectiveness of Abrocitinib for Patients Aged 12 and Older with Moderate to Severe Atopic Dermatitis
SpainStudy on Amlitelimab for Patients Aged 12 and Older with Moderate-to-Severe Atopic Dermatitis
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