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PKANPantothenate Kinase-Associated Neurodegeneration
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(NASDAQ: RTRX) today announced that the Phase 3 FORT Study evaluating the safety and efficacy of fosmetpantotenate compared to placebo in patients with pantothenate kinase-associated neurodegeneration (PKAN) did not meet its primary endpoint and did not demonstrate a difference between treatment groups.
At present, there is no specific treatment for PKAN and the management is symptomatic.
Deep brain stmulation was less effective in the birth injury group as compared to the three most favorable groups: DYT1, PKAN, and tardive dystonia.
Missense mutations (majority of identified variants) can lead to either early or late onset PKAN forms.
One study analyzed brain MRIs of 26 patients with genetically confirmed PKAN, 21 patients with neuroferritinopathy, 10 patients with aceruloplasminemia, and four patients with NBIA type 2.
Biopharmaceutical company Retrophin (Nasdaq:RTRX) said on Thursday that it has entered into an agreement with the US Food and Drug Administration (FDA) under the Special Protocol Assessment (SPA) process for a Phase 3 clinical trial evaluating RE-024 for the treatment of pantothenate kinase-associated neurodegeneration (PKAN), a rare, genetic, and life-threatening neurological disorder characterised by a host of progressively debilitating symptoms that typically begin in early childhood.
PKAN is classified as classic or atypical according to the age of onset, rate of progression and severity of the motor symptoms [3].
(NASDAQ: RTRX) disclosed that its Phase 3 FORT study of fosmetpantotnate in patients with PKAN did not meet its primary or secondary endpoints.
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.
Upon review of the available safety and tolerability data of fosmetpantotenate in adult patients with PKAN in the study to date, the DMC recommended that the pivotal trial continue as planned, and supported initiation of enrollment in pediatric patients aged 6 to 17.
M2 PHARMA-November 11, 2016-Retrophin reaches agreement with US FDA to evaluate RE-024 in PKAN patients
Leigh's disease 13 MLD 4 Wilson's disease 2 Gangliosidosis 2 PKAN (NBIA Type 1) 2 Canavan's disease 1 Alexander's disease 1 Glutaric aciduria 1 The criterion of image interpretation was the image based classification, which grossly divides these disorders on the pattern of involvement of gray and white matter.