NIEMANN-PICK DISEASE TYPE C PREVENTIVE AND/OR THERAPEUTIC AGENT

PROBLEM TO BE SOLVED: To provide a pharmaceutical composition that has the action to improve decreases in the stability of NPC1 mutant and folding efficiency and is useful for the prevention and/or treatment of Niemann-Pick disease type C.SOLUTION: A Niemann-Pick disease type C preventive and/or the...

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Hauptverfasser: OGANE KENJI, SODEOKA MIKIKO
Format: Patent
Sprache:eng ; jpn
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Zusammenfassung:PROBLEM TO BE SOLVED: To provide a pharmaceutical composition that has the action to improve decreases in the stability of NPC1 mutant and folding efficiency and is useful for the prevention and/or treatment of Niemann-Pick disease type C.SOLUTION: A Niemann-Pick disease type C preventive and/or therapeutic agent comprises a compound represented by formula (I), its ester or salt as an active ingredient (Ar-Arindependently represent a substituted/unsubstituted aromatic group; Xis CHor O; preferably Aris 4-alkyl-1,2,4-triazole-5-one group).SELECTED DRAWING: Figure 2D 【課題】NPC1変異体の安定性及びフォールディング効率の低下を改善する作用を有し、ニーマンピック病C型の予防及び/又は治療のために有用な医薬組成物の提供。【解決手段】式(I)で示される化合物、そのエステル又は塩を有効成分として含有するニーマンピック病C型の予防及び/又は治療剤。(Ar1〜Ar3は各々独立に置換/非置換の芳香族基;X1はCH2又はO;好ましくはAr3が4−アルキル−1,2,4−トリアゾール−5−オン基)【選択図】図2D