JP2012006953 TREATMENT OF GLYCOGEN STORAGE DISEASE TYPE II

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Application Number 2011173569

Application Date 09.08.2011

Publication Number 2012006953

Publication Date 12.01.2012

Publication Kind A

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Applicants DUKE UNIV
デューク　ユニバーシティ

Inventors CHEN YUAN-TSONG
チェン，ユアン－ツォン

Agents 小野　新次郎
小林　泰
千葉　昭男
富田　博行
泉谷　玲子

Priority Data 60/219,237 18.07.2000 US

Title

(EN) TREATMENT OF GLYCOGEN STORAGE DISEASE TYPE II

(JA) ＩＩ型糖原病の治療

Abstract

(EN)

PROBLEM TO BE SOLVED: To provide a composition for use in treatment of glycogen storage disease type II.

SOLUTION: There is provided a pharmaceutical composition comprising human acid α-glucosidase in a container with a label containing instructions for administration of the composition for treatment of glycogen storage disease type II. The human acid α-glucosidase is preferably recombinant human acid α-glucosidase, further preferably a precursor of recombinant human acid α-glucosidase. Further more preferably, the precursor of recombinant human acid α-glucosidase is produced in Chinese hamster ovary cells. The human acid α-glucosidase is administered at the regular interval (for example, monthly, bimonthly, weekly, twice weekly, daily). In a preferred embodiment, the human acid α-glucosidase is administered intravenously, intramuscularly, intrathecally or intraventricularly.

(JA)

【課題】II型糖原病を有する個体を治療するための医薬組成物の提供。
【解決手段】II型糖原病の治療用組成物の投与のための指示書を含むラベルを有する容器内に、ヒト酸α−グルコシダーゼを含有する医薬組成物。該ヒト酸α−グリコシダーゼは、好ましくは組換えヒト酸α−グリコシダーゼ、より好ましくはヒト酸α−グリコシダーゼの前駆体形態、およびさらにより好ましくはチャイニーズハムスター卵巣細胞において産生されるヒト酸α−グリコシダーゼの前駆体形態である。該ヒト酸α−グリコシダーゼは、周期的（例えば、毎月、月に２回、毎週、週に２回、毎日）に投与される。好ましい態様では、該ヒト酸α−グリコシダーゼは、静脈内；筋肉内；髄腔内；または脳室内に投与される。
【選択図】なし

A	HUMAN NECESSITIES
61	MEDICAL OR VETERINARY SCIENCE; HYGIENE
K	PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
38	Medicinal preparations containing peptides
16	Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
43	Enzymes; Proenzymes; Derivatives thereof
46	Hydrolases (3)

A	HUMAN NECESSITIES
61	MEDICAL OR VETERINARY SCIENCE; HYGIENE
J	CONTAINERS SPECIALLY ADAPTED FOR MEDICAL OR PHARMACEUTICAL PURPOSES; DEVICES OR METHODS SPECIALLY ADAPTED FOR BRINGING PHARMACEUTICAL PRODUCTS INTO PARTICULAR PHYSICAL OR ADMINISTERING FORMS; DEVICES FOR ADMINISTERING FOOD OR MEDICINES ORALLY; BABY COMFORTERS; DEVICES FOR RECEIVING SPITTLE
1	Containers specially adapted for medical or pharmaceutical purposes
05	for collecting, storing or administering blood, plasma or medical fluids
14	Details; Accessories therefor

A	HUMAN NECESSITIES
61	MEDICAL OR VETERINARY SCIENCE; HYGIENE
P	SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
3	Drugs for disorders of the metabolism
08	for glucose homeostasis
10	for hyperglycaemia, e.g. antidiabetics

A	HUMAN NECESSITIES
61	MEDICAL OR VETERINARY SCIENCE; HYGIENE
K	PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
38	Medicinal preparations containing peptides
16	Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
43	Enzymes; Proenzymes; Derivatives thereof
46	Hydrolases (3)

A	HUMAN NECESSITIES
61	MEDICAL OR VETERINARY SCIENCE; HYGIENE
J	CONTAINERS SPECIALLY ADAPTED FOR MEDICAL OR PHARMACEUTICAL PURPOSES; DEVICES OR METHODS SPECIALLY ADAPTED FOR BRINGING PHARMACEUTICAL PRODUCTS INTO PARTICULAR PHYSICAL OR ADMINISTERING FORMS; DEVICES FOR ADMINISTERING FOOD OR MEDICINES ORALLY; BABY COMFORTERS; DEVICES FOR RECEIVING SPITTLE
1	Containers specially adapted for medical or pharmaceutical purposes
05	for collecting, storing or administering blood, plasma or medical fluids
14	Details; Accessories therefor

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1. JP2012006953 - TREATMENT OF GLYCOGEN STORAGE DISEASE TYPE II

C	CHEMISTRY; METALLURGY
12	BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
Y	ENZYMES
302	Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
01	Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
0102	Alpha-glucosidase (3.2.1.20)