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A B C D E F G H I J K L M N O P Q R S T U V W X Y ZClinical Trials for Rare Diseases
- A Study of SGT-212 Gene Therapy in Friedreich's Ataxia
- Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)
- Ascending Doses of Crofelemer Powder for Oral Solution in Pediatric Microvillus Inclusion Disease (MVID)
- A First-in-human Study of KK8123 in Adults With X-linked Hypophosphatemia
- CTH120 First-in-Human Study: Single and Multiple Ascending Doses and Potential Food Interaction
- Fluoxetine in KCNC1-related Disorder
- An Extension Study to Evaluate the Long-Term Safety and Efficacy of PTC518 in Participants With Huntington's Disease (HD)
- Cannabidiol in Children and Young Adults With Rare Disease-associated Severe Epilepsy
- A Study to Evaluate Emapalumab in Japanese Healthy Volunteers.
- Severe Bullous Drug Eruption and Filgrastim
- Burosumab in Children and Adolescents With X-linked Hypophosphatemia
- Study of A166 in Patients With Relapsed/Refractory Cancers Expressing HER2 Antigen or Having Amplified HER2 Gene
- Vigil + Irinotecan and Temozolomide in Ewing's Sarcoma
- Durvalumab in Combination With Tremelimumab in Subjects With Advanced Rare Solid Tumors
- EDS in Ataxia Telangiectasia Patients
- Targeted Busulfan, Fludarabine Conditioning Regimen for Hematopoietic Stem Cell Transplantation in GRD
- Phase II Study of Imatinib Mesylate in Patients With Life Threatening Malignant Rare Diseases