p.204 抽出テキスト

用し個々の患者の症状にあわせた指導を行い,対照と比較し症状やQOL の改善を評価する試みである.今後の治療評価とともに患者に特化したIT 技術の利用が期待される.文献1)  Waldhuter N, Köhler W, Hemmati PG, et al: Allogeneic hematopoietic stem cell

transplantation with myeloablative conditioning for adult cerebral X-linked adrenoleukodystrophy. J Inherit Metab Dis 2019; 42: 313-324. 2)  Matsukawa T, Yamamoto T, Honda A, et al: Clinical efficacy of haematopoietic stem

cell transplantation for adult adrenoleukodystrophy. Brain Commun 2020; 2: fcz048. 3)  Weinhofer I, Rommer P, Zierfuss B, et al: Neurofilament light chain as a potential

biomarker for monitoring neurodegeneration in X-linked adrenoleukodystrophy. Nat Commun 2021; 12: 1816. 4)  Kakumoto T, Matsukawa T, Ishiura H, et al: Neurofilament light chain levels in

cerebrospinal fluid as a sensitive biomarker for cerebral adrenoleukodystrophy. Ann Clin Transl Neurol 2023; 10: 1230-1238. 5)  Huffnagel IC, van Ballegoij WJC, Vos JMBW, et al: Longitudinal diffusion MRI as

surrogate outcome measure for myelopathy in adrenoleukodystrophy. Neurology 2019; 93: e2133-e2143. 6)  Casasnovas C, Ruiz M, Schlüter A, et al: Biomarker identification, safety, and

efficacy of high-dose antioxidants for adrenomyeloneuropathy: a Phase II Pilot Study. Neurotherapeutics 2019; 16: 1167-1182. 7)  Monternier PA, Singh J, Parasar P, et al: Therapeutic potential of deuterium-

stabilized(R)-pioglitazone-PXL065-for X-linked adrenoleukodystrophy. J Inherit Metab Dis 2022; 45: 832-847. 8)  Rodríguez-Pascau L, Vilalta A, Cerrada M, et al: The brain penetrant PPARγ agonist

leriglitazone restores multiple altered pathways in models of X-linked adrenoleukodystrophy. Sci Transl Med 2021; 13: eabc0555. 9)  Köhler W, Engelen M, Eichler F, et al: Safety and efficacy of leriglitazone for

preventing disease progression in men with adrenomyeloneuropathy(ADVANCE): a randomised, double-blind, multi-centre, placebo-controlled phase 2-3 trial. Lancet Neurol 2023; 22: 127-136. 10)  Golse M. Weinhofer I, Blanco B, et al: Leriglitazone halts disease progression in

adult patients with early cerebral adrenoleukodystrophy. Brain 2024; 147: 3344-

3351 11)  van Ballegoij WJC, van de Stadt SIW, Huffnagel IC, et al: Postural Body Sway as

Surrogate Outcome for Myelopathy in Adrenoleukodystrophy. Front Physiol 2020; 11: 786. 12)  van Ballegoij WJC, Huffnagel IC, van de Stadt SIW, et al: Optical coherence