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Genetic Testing Identified Causative Variants in Nearly 20% of Early-Onset Bilateral Cataracts Cases

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Long-Term Efficacy and Safety of Sparsentan in Young Patients WithFSGS: 240-Week Analysis of the DUET Open-Label Extension (OLE)

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Sparsentan Publications 2023

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Population-based Incidence Estimates of Classical Homocystinuria Using the Genome Aggregation Database (gnomAD)

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A Longitudinal Study of Cognitive Function in Classical Homocystinuria (HCU) Demonstrates Distinct Deficits in Inhibitory Control

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Insights from the First Genetic Evaluation of a Longitudinal Natural History Study in Classical Homocystinuria (HCU)

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Posters HCU

Population-based Incidence Estimates of Classical Homocystinuria Using the Genome Aggregation Database (gnomAD)

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Posters HCU

A Longitudinal Study of Cognitive Function in Classical Homocystinuria (HCU) Demonstrates Distinct Deficits in Inhibitory Control

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Posters HCU

Insights from the First Genetic Evaluation of a Longitudinal Natural History Study in Classical Homocystinuria (HCU)

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Pegtibatinase, an Investigational Enzyme Replacement Therapy for the Treatment of Classical Homocystinuria: Initial Results From the Phase 1/2 COMPOSE Study

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Pegtibatinase, an Investigational Enzyme Replacement Therapy for the Treatment of Classical Homocystinuria: Initial Results from the Phase 1/2 COMPOSE Study

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Dietary Goals and Current Challenges in the Management of Classical Homocystinuria: Insights From Multinational Real-World Experience

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Sparsentan, the dual endothelin and angiotensin receptor antagonist (DEARA), improves kidney function and lifespan and protects against hearing loss in Alport mice with developed renal structural changes

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