Genetic Disorder
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Sign up freeTop drugs studied for this disease
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CLINICAL_TRIALS research · 2026-02-05Clinical Trial Update: Neonatal Seizure Registry, GEnetics of Post-Neonatal Epilepsy
A clinical trial registry entry from University of California, San Francisco describing a Neonatal Seizure Registry and Genetics of Post-Neo…
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CLINICAL_TRIALS trial_result · 2021-01-26Clinical Trial Update: Fludarabine Phosphate, Cyclophosphamide, and Total-Body Irradiation Followed by Donor Bone Marrow Transplant and Cyclophosphamide, Mycophenolate Mofetil, Tacrolimus, and Sirolimus in Treating Patients With Primary Immunodeficiency Disorders or Noncancerous Inherited Disorders [Phase 2]
Phase 2 trial evaluating a conditioning regimen followed by donor bone marrow or stem cell transplant in patients with primary immunodeficie…
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CLINICAL_TRIALS trial_result · 2019-11-04Clinical Trial Update: Pharmacokinetics of Thymoglobulin in Paediatric Haematopoietic Stem-cell Transplants [Phase 2]
Phase 2 trial updating pharmacokinetics of Thymoglobulin in pediatric hematopoietic stem-cell transplants; study terminated with results pos…
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CLINICAL_TRIALS trial_result · 2019-10-14Clinical Trial Update: Investigating the Long-term Efficacy and Safety of Two Doses of NN-220 (Somatropin) in Short Stature Due to Noonan Syndrome [Phase 3]
Phase 3 trial assessing long-term efficacy and safety of two doses of NN-220 (somatropin) in short stature due to Noonan syndrome has comple…