Hypometabolism and atrophy patterns associated with Niemann-Pick type C.

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Tác giả: Pablo Aguiar, Manuel Arias, Cristina Castro, Julia Cortés, Jordi Gascon-Bayarri, Michel J Grothe, Alexis Moscoso, Virginia Pubul, Gabriel Reynes-Llompart, Laura Rodríguez-Bel, Jesús Silva-Rodríguez, María Jesús Sobrido

Ngôn ngữ: eng

Ký hiệu phân loại: 646.407 Patterns

Thông tin xuất bản: Germany : EJNMMI research , 2025

Mô tả vật lý:

Bộ sưu tập: NCBI

ID: 680967

 BACKGROUND: Niemann-Pick disease type C (NP-C) is a rare genetic lysosomal lipid storage disorder characterized by progressive neurological impairment. Early diagnosis is critical for initiating treatment with miglustat, which can decelerate disease progression. In this study, we evaluated a cohort of 22 NP-C patients who underwent MRI, [ RESULTS: Group-level MRI analysis identified significant cerebellar and thalamic atrophy (d = 1.56, p <
  0.0001 and d = 1.09, p <
  0.001, respectively), with less pronounced involvement of the frontal lobe and hippocampus, which aligned with existing neuropathological understanding and guidelines. Conversely, [ CONCLUSIONS: Our findings delineate a discernible hypometabolism pattern specific to NP-C that distinguishes it from other neurodegenerative conditions, thus suggesting that [ TRIAL REGISTRATION: XUNTA 2015/140. Registered 21 April 2015.
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