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Tagged: Neurodegeneration

PKAN drug fails Phase III trial: How to avoid a similar disaster.

November 26th, 2019 | Neurodegeneration

Retrophin’s experimental PKAN drug fosmetpantotenate fails phase III trial Pantothenate kinase-associated neurodegeneration (PKAN) is a rare disease characterized by a progressive neurodegenerative disorder and buildup of iron in the brain which is estimated to affect up to 5000 patients worldwide. PKAN is inherited in an autosomal recessive manner and is caused by mutations in the […]

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