Use of aprenomor in treatment of type C Niemann-Pick Disease in patients with ER-type missense mutations

Use of aprenomor in treatment of type C Niemann-Pick Disease in patients with ER-type missense mutations

The present invention relates to an active pharmaceutical ingredient selected from the group consisting of N-[2-hydroxy-3-(1-piperidinyl)-propoxy]-pyridine-1-oxide-3-methylenimine acyl chloride, stereoisomers thereof, and acid addition salts thereof; in particular aprenomos for use in a method of tr...

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Bibliographische Detailangaben
Hauptverfasser: JENSEN THOMAS KIRKEGAARD, BLATTLER THOMAS, FREIBURGER, LAURA, C, AIDALI CRISTINA, PETERSEN NIKOLAJ HAVNSTED TORP
Format: Patent
Sprache:chi ; eng
Schlagworte:
HUMAN NECESSITIES
HYGIENE
MEDICAL OR VETERINARY SCIENCE
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
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Beschreibung
Zusammenfassung:The present invention relates to an active pharmaceutical ingredient selected from the group consisting of N-[2-hydroxy-3-(1-piperidinyl)-propoxy]-pyridine-1-oxide-3-methylenimine acyl chloride, stereoisomers thereof, and acid addition salts thereof; in particular aprenomos for use in a method of treating Niemann-Pickup Disease type C (NPC) wherein the patient has an endoplasmic reticulum (ER) type missense mutation in the NPC gene. 本发明涉及选自N-[2-羟基-3-(1-哌啶基)-丙氧基]-吡啶-1-氧化物-3-甲亚胺酰氯、其立体异构体及其酸加成盐的活性药物成分；具体为阿瑞洛莫，用于治疗C型尼曼皮克病(NPC)的方法中，其中患者在NPC基因中具有内质网(ER)型错义突变。