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Retinitis Pigmentosa Syndrome
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Retinitis Pigmentosa Syndrome trials may be worth asking aboutClinical trial pipeline · Data from ClinicalTrials.gov
See which Retinitis Pigmentosa Syndrome trials may be worth asking aboutNorth America
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Background: Pyrimidine and purine metabolism disorders (DPPMs) affect how the body metabolizes chemicals called pyrimidines and purines. DPPMs can cause dysfun…
The main goal of the project is provision of a global registry for mitochondrial disorders to harmonize previous national registries, enable world-wide particip…
Study OpCT-001-101 is a Phase 1/2a first-in-human, multisite, 2-part interventional study to evaluate the safety, tolerability, and the effect on clinical outco…
This study will evaluate the use of autologous bone marrow derived stem cells (BMSC) for the treatment of retinal and optic nerve damage or disease.
Bardet-Biedl syndrome (BBS) and other rare disorders associated with impairment of the melanocortin-4 receptor (MC4R) pathway are characterized by severe early-…
Hepato-renal fibrocystic diseases (HRFD) is a term developed that encompasses rare diseases such as Autosomal Recessive Polycystic Kidney Disease (ARPKD), and o…
Objective: One objective of this study is to genetically map and identify mutated genes for human hereditary hearing loss. A second objective is to study the fu…
This is a Phase 1/2 Study to Assess the Safety and Efficacy of OCU400 in patients with retinitis pigmentosa associated with NR2E3 and RHO mutations and in patie…