Retrophin drug to treat nervous system disorder fails late-stage study [Reuters]
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Source: Reuters
Retrophin drug to treat nervous system disorder fails late-stage study - Reuters 1 Min Read (Reuters) - Retrophin Inc said on Thursday its treatment for a rare neurological disorder failed to meet the main goal of improving patient’s ability to conduct daily activities in a late-stage study. The treatment, fosmetpantotenate, was being tested in 84 patients with pantothenate kinase-associated neurodegeneration, a rare, genetic and life-threatening disorder. The condition, which can lead to movement disorders and visual impairment, is estimated to affect up to 5,000 people worldwide, Retrophin said. The study analyzed fosmetpantotenate using a scale to measure improvement in daily activity in patients called the PKAN-ADL scale through a period of 24 weeks. The company said fosmetpantotenate was found to be safe and well-tolerated and that it would work closely with the study investigators to further analyze the results. The treatment, tested against placebo, also failed to meet its seco
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