A healthy volunteer phase 1 study of RE-024, a potential phosphopantothenate replacement therapy for patients with pantothenate kinase-associated neurodegeneration (PKAN)
Objective: To assess the safety, tolerability, and pharmacokinetics of single oral doses of RE-024 in healthy adult volunteers. Background: PKAN is a rare, autosomal recessive,…RE-024: A potential phosphopantothenate replacement therapy in 2 patients with pantothenate kinase-associated neurodegeneration (PKAN)
Objective: To evaluate the safety and potential efficacy of RE-024 in open treatment of 2 patients with PKAN. Background: PKAN is an autosomal recessive, neurodegenerative…
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