Kyowa Medex Co., Ltd.

The United States of America As Represented by the Secretary of

Kumamoto University

Tetsumi Irie

Kaneto Uekama

Hiroyuki Sugiuchi

Eiko Shutoh

Kazuhito Miyauchi

Susumu Ohsawa

Akira Miike

Toshio Tatano

Norihiko Kayahara

Josef Pitha

Takumi Era

Juan J Torres-Labandeira

Provided is a pharmaceutical composition for the treatment of disorders such as Niemann-Pick disease and GM1 gangliosidosis which are caused by the storage of cholesterol, such as lysosomal storage disease. Also provided is a method for screening for said pharmaceutical compositions that uses iPS cell strains that phenocopy phentotypes of these disorders. Provided is a pharmaceutical composition for the treatment and/or prevention of lysosomal storage disease, characterized by containing hydroxypropyl-&#x3b3;-cyclodextrin as an active ingredient. Also provided are an iPS cell strain derived from patients suffering from intractable disorders and prepared using a new temperature-sensitive Sendai virus vector, and a screening method for pharmaceuticals using said iPS cell strain.

Drug for the treatment of cholesterol accumulation disorders

Method for determination of cholesterol in low-density lipoprotein or

Provided is a method of determining the amount of cholesterol in high-density lipoprotein (HDL), which comprises reacting an HDL-containing sample with cholesterol esterase and cholesterol oxidase or cholesterol dehydrogenase in the presence of a reagent for aggregating lipoproteins- except HDL, and determining the amount of hydrogen peroxide or reductive co-enzyme formed therein.

Method of determining the amount of cholesterol in a high-density

The dissolution of lipophilic compounds in aqueous solutions of hydroxypropylcyclodextrins can be accelerated by the addition of co-solubilizers, such as ethanol or ammonia, which again can be removed, together with water, by evaporation or by freeze-drying leaving lipophile: hydroxypropylcyclodextrin complexes as a residue. The co-solubilizer method was used successfully with steroid drugs (5-androstene-3.beta.,17.beta.-diol, 4-androstene-3,17-dione, dehydroepiandrosterone, dexamethasone, 5-.alpha.-dihydro-testosterone, 6-methylprednisolone, and testosterone), peptides (gramicidin S) and a macrocyclic antibiotic (amphotericin B). The complexes prepared in this manner were amorphous and possessed satisfactory stability.

Preparation of lipophile:hydroxypropylcyclodextrin complexes by a method

Growing community of inventors

Kumamoto, Japan

Tetsumi Irie