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Gene Therapy Clinical Trial for the Treatment Of Leber's HereDitary Optic Neuropathy

The objective of this clinical study is to select the optimal dose and evaluate the safety and efficacy of NR082 in treatment of LHON caused by mitochondrial ND4 gene mutation....

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A Study of TAK-771 in Japanese Participants With Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP) and Multifocal M...

The main aim of the study is to check for side effects from TAK-771, and to check how well TAK-771 controls symptoms in Japanese participants with chronic inflammatory demyelinating polyradiculoneuropathy...

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Assess Safety and Tolerability of ART-123 + FOLFOX + Bevacizumab in Metastatic Colorectal Cancer Patients

To evaluate the safety and tolerability of ART-123 in patients with metastatic colorectal cancer who receive oxaliplatin-containing chemotherapy and bevacizumab

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A Study of DB-OTO, an AAV Based Gene Therapy, in Children/Infants With Hearing Loss Due to Otoferlin Mutations

This is a first-in-human, multicenter, Phase 1/2, open-label, 2-part trial with a single-ascending dose patient cohort (Part A) and a bilateral expansion patient cohort (Part B) to evaluate the safety,...