Clinical Trials
- Brain, Spinal Cord & Nervous System, Genetics
A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy (MIT-E)
- Ages18 years and younger
- GenderBoth
- Endocrine System, Genetics
Compassionate Use Program for Olipudase Alfa Enzyme Replacement Therapy for Patients With Chronic Acid Sphingomyelinase Deficiency (ASMD)
- Ages3 years and older
- GenderBoth
- Children's Health, Genetics
Pediatric Genomics Discovery Program (PGDP)
- GenderBoth