The FOsmetpantotenate Replacement Therapy (FORT) Pivotal Trial: Utilization of a Novel Primary Efficacy Outcome in Patients with Pantothenate Kinase-Associated Neurodegeneration
Objective: To describe the methodology and novel primary efficacy outcome measure in the ongoing pivotal FORT trial in patients with pantothenate kinase-associated neurodegeneration (PKAN). Background:…Development of a Clinical Outcomes Assessment (COA) in Pantothenate-Kinase Associated Neurodegeneration (PKAN): Item Generation and Clinimetric Properties
Objective: To develop a clinical outcome assessment (COA) scale that measures key aspects of functioning and daily living in patients with pantothenate kinase-associated neurodegeneration (PKAN)…Neurodegeneration with brain iron accumulation (NBIA): Two cases with different subtypes and a rare mutation
Objective: We present two cases with clinical, radiological and genetical (a very rare mutation) findings of neurodegeneration with brain iron accumulation (NBIA). Results: Case 1.…Pantotenate kinase associated neurodegeneration (PKAN): Proposal for a clinical rating scale
Objective: To design and validate a quantitative method for the assessment of PKAN patients. Background: Despite advances in the research of novel therapies for PKAN,…RE-024: Mechanism of action and efficacy in non-clinical models
Objective: To confirm the mechanism of action of RE-024, and evaluate its activity in human cellular models and brain penetration in animal models. Background: Pantothenate…Development of a human neuroblastoma model of pantothenate kinase-associated neurodegeneration
Objective: To establish a stable human neuronal model defective in PanK2. Background: Pantothenate Kinase-Associated Neurodegeneration (PKAN), is caused by mutations in PANK2, which encodes one…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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