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PKANPantothenate Kinase-Associated Neurodegeneration
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The Company's approach centers on its pipeline featuring late-stage assets targeting rare diseases with significant unmet medical needs, including fosmetpantotenate for pantothenate kinase-associated neurodegeneration (PKAN), a life-threatening neurological disorder that typically begins in early childhood, and sparsentan for focal segmental glomerulosclerosis (FSGS) and IgA nephropathy (IgAN), disorders characterized by progressive scarring of the kidney often leading to end-stage renal disease.
Involvement of globus pallidus and midbrain nuclei in pantothenate kinase-associated neurodegeneration: measurement of T2 and T2* time.
Beconi, "Development of a human neuroblastoma model of pantothenate kinase-associated neurodegeneration," in Proceedings of the ACMG Annual Clinical Genetics Meeting, Tampa, Fla, USA, March 2016, http://epostersonline .com/acmg2016/node/2740.
According to the company, the primary endpoint will be the change in score on the Pantothenate Kinase-associated Neurodegeneration Activities of Daily Living (PKAN-ADL) scale, from baseline through 24 weeks of treatment.
Yuen, "Pantothenate kinase-associated neurodegeneration in two Chinese children: identification of a novel PANK2 gene mutation, " Hong Kong Medical Journal, vol.
The Phase 3 FORT Study evaluating fosmetpantotenate for the treatment of pantothenate kinase-associated neurodegeneration (PKAN) remains on-track to report top-line results in the third quarter of 2019, followed by anticipated submissions for a New Drug Application (NDA) in the U.S.