If the NDA files Q4 2026 and standard review applies, expect an FDA decision around Q4 2027
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Results Establishing transgenic zebrafish models for LMNA -related muscular laminopathy To investigate the pathogenesis of LMNA -related muscular laminopathy, we created transgenic zebrafish lines expressing human wild-type LMNA and five LMNA mutants
Imaging demonstrates various manifestations, including diffuse abnormalities in the supratentorial white matter, reduced brain volume, diffuse cerebral edema, basal ganglia calcifications, and delayed myelination [78, 79]