DK2083811T3 - Fremgangsmåder til behandling af downs syndrom, fragilt x-syndrom og autisme - Google Patents

Fremgangsmåder til behandling af downs syndrom, fragilt x-syndrom og autisme Download PDF

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DK2083811T3
DK2083811T3 DK07862184.4T DK07862184T DK2083811T3 DK 2083811 T3 DK2083811 T3 DK 2083811T3 DK 07862184 T DK07862184 T DK 07862184T DK 2083811 T3 DK2083811 T3 DK 2083811T3
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baclofen
composition
use according
agonist
mice
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Randall L Carpenter
Kathryn Roberts
Mark F Bear
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Clinical Res Ass Llc
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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/185Acids; Anhydrides, halides or salts thereof, e.g. sulfur acids, imidic, hydrazonic or hydroximic acids
    • A61K31/19Carboxylic acids, e.g. valproic acid
    • A61K31/195Carboxylic acids, e.g. valproic acid having an amino group
    • A61K31/197Carboxylic acids, e.g. valproic acid having an amino group the amino and the carboxyl groups being attached to the same acyclic carbon chain, e.g. gamma-aminobutyric acid [GABA], beta-alanine, epsilon-aminocaproic acid or pantothenic acid
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/185Acids; Anhydrides, halides or salts thereof, e.g. sulfur acids, imidic, hydrazonic or hydroximic acids
    • A61K31/19Carboxylic acids, e.g. valproic acid
    • A61K31/195Carboxylic acids, e.g. valproic acid having an amino group
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/21Esters, e.g. nitroglycerine, selenocyanates
    • A61K31/215Esters, e.g. nitroglycerine, selenocyanates of carboxylic acids
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/41Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with two or more ring hetero atoms, at least one of which being nitrogen, e.g. tetrazole
    • A61K31/425Thiazoles
    • A61K31/428Thiazoles condensed with carbocyclic rings
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P15/00Drugs for genital or sexual disorders; Contraceptives
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/08Antiepileptics; Anticonvulsants
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/18Antipsychotics, i.e. neuroleptics; Drugs for mania or schizophrenia
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/22Anxiolytics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00

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  • General Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Veterinary Medicine (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Reproductive Health (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Ultra Sonic Daignosis Equipment (AREA)
  • Heat Treatment Of Sheet Steel (AREA)
  • Surgical Instruments (AREA)

Claims (30)

1. Sammensætning til anvendelse ved forbedring af mindst ét element valgt fra gruppen bestående af et socialt domæne og et kommunikationsdomæne hos et menneske med autisme, hvor sammensætningen indbefatter en terapeutisk effektiv mængde af mindst én gamma-aminosmørsyre-agonist.
2. Sammensætning til anvendelse ved forbedring af mindst ét element valgt fra gruppen bestående af et socialt domæne og et kommunikationsdomæne hos et menneske med autismespektrumforstyrrelse, hvor sammensætningen indbefatter en terapeutisk effektiv mængde af mindst én gamma-aminosmørsyre-agonist.
3. Sammensætning til anvendelse ved behandling af et menneske med fragilt X-syndrom, hvor sammensætningen indbefatter en terapeutisk effektiv mængde af mindst én gamma-aminosmørsyre-agonist.
4. Sammensætning til anvendelse ved behandling af et menneske med Downs syndrom, hvor sammensætningen indbefatter en terapeutisk effektiv mængde af mindst én gamma-aminosmørsyre-agonist.
5. Sammensætning til anvendelse ved forbedring af et socialt domæne hos et menneske med autisme, hvor sammensætningen indbefatter en terapeutisk effektiv mængde af mindst én gamma-aminosmørsyre-agonist.
6. Sammensætning til anvendelse ifølge et hvilket som helst af de foregående krav, hvorgamma-aminosmørsyre (GABA)-agonisten indbefatter en GABA(B)-agonist.
7. Sammensætning til anvendelse ifølge krav 3 eller krav 6, hvor GABA(B)-agonisten indbefatter baclofen, fortrinsvis R-baclofen.
8. Sammensætning til anvendelse ifølge krav 7, hvor baclofenet er mindst ca. 80 molprocent R-baclofen, mindst ca. 85 molprocent R-baclofen, mindst ca. 90 molprocent R-baclofen, mindst ca. 95 molprocent R-baclofen eller mindst ca. 9£ molprocent R-baclofen.
9. Sammensætning til anvendelse ifølge et hvilket som helst af de foregående krav, hvilken anvendelse yderligere indbefatter administration til mennesket af mindst ét element valgt fra gruppen bestående af et antidepressivum, en a2-adrenerg agonist, et antikonvulsivum, en nicotinreceptoragonist, en endocannabinoidreceptoragonist, et antikonvulsivum, et antipsykotikum, en AMPA-agonist og en M1-muscarinreceptorantagonist.
10. Sammensætning til anvendelse ifølge krav 2 eller krav 5, hvor mennesket yderligere har fragilt X-syndrom.
11. Sammensætning til anvendelse ifølge et hvilket som helst af de foregående krav, hvilken anvendelse yderligere indbefatter administration af mindst én gruppe 1-mGluR-antagonist til mennesket.
12. Sammensætning til anvendelse ifølge krav 11, hvor gruppe 1-mGluR-antagonisten enten er en mGluR5-antagonist eller en mGluRI-antagonist.
13. Sammensætning til anvendelse ifølge et hvilket som helst af de foregående krav, hvilken anvendelse yderligere indbefatter en forbedring af en kognitiv funktion hos mennesket.
14. Sammensætning til anvendelse ifølge krav 13, hvor en forbedring af den kognitive funktion er en forbedring af mindst ét element valgt fra gruppen bestående af hukommelse, indlæring, opmærksomhed, eksekutiv funktion, informationsbehandling, begrebsdannelse, årvågenhed og problemløsning.
15. Sammensætning til anvendelse ifølge krav 1 eller krav 2, hvor sammensætningen indbefatter en terapeutisk effektiv mængde med mindst ca. 90 molprocent R-baclofen i forhold til det totale R-baclofen og S-baclofen i sammensætningen.
16. Sammensætning til anvendelse ifølge et hvilket som helst af de foregående krav, hvor forbedringen af det sociale domæne hos mennesket yderligere indbefatter reduktion af mindst ét element valgt fra gruppen bestående af en obsessiv-kompulsiv tilstand, en sensorisk overfølsomhed, en angstlidelse, en krampeanfaldslidelse, irritabilitet, aggression, skrækudløst tremor, repetitiv/selvstimulerende adfærd, vigende øjenkontakt, håndbidning, hovedbanken og slag mod andre.
17. Sammensætning til anvendelse ifølge et hvilket som helst af de foregående krav, hvor forbedringen af det sociale domæne hos mennesket indbefatter forbedring af mindst ét element valgt fra gruppen bestående af socialisering, en passende reaktion på talesprog, spontane forsøg på interaktion, øjenkontakt, humør, motivation og ro.
18. Sammensætning til anvendelse ifølge et hvilket som helst af kravene 1,2 eller 5 eller krav, der afhænger deraf, hvilken anvendelse yderligere indbefatter en forbedring af et kommunikationsdomæne hos mennesket.
19. Sammensætning til anvendelse ifølge krav 18, hvor forbedringen af kommunikationsdomænet indbefatter en forbedring af det verbale sprog.
20. Sammensætning til anvendelse ifølge et hvilket som helst af de foregående krav, hvor sammensætningen er beregnet til at reducere mindst ét element valgt fra gruppen bestående af irritabilitet og aggression hos et menneske med autisme.
21. Sammensætning til anvendelse ifølge et hvilket som helst af de foregående krav, hvorgamma-aminosmørsyre-agonisten, GABA(B)-agonisten, baclofenet eller R-baclofenet administreres oralt til mennesket.
22. Sammensætning til anvendelse ifølge et hvilket som helst af de foregående krav, hvorgamma-aminosmørsyre-agonisten, baclofenet eller R-baclofenet administreres til mennesket i en dosis på ca. 2,5 mg, ca. 5 mg, ca. 10 mg, ca. 15 mg, ca. 20 mg, ca. 25 mg, ca. 30 mg, ca. 40 mg, ca. 45 mg, ca. 50 mg eller ca. 100 mg.
23. Sammensætning til anvendelse ifølge krav 22, hvor gamma-aminosmørsyre-agonisten, baclofenet eller R-baclofenet administreres en enkelt daglig dosis eller i to daglige doser eller tre daglige doser eller fire daglige doser eller fem daglige doser eller i flere daglige doser.
24. Sammensætning til anvendelse ifølge et hvilket som helst af de foregående krav, hvor mindst ét af et socialt domæne, et kommunikationsdomæne og et kognitivt domæne forbedres hos mennesket efter behandling med gamma-aminosmørsyre-agonisten.
25. Sammensætning til anvendelse ifølge et hvilket som helst af de foregående krav, hvor det sociale domæne hos mennesket forbedres.
26. Sammensætning til anvendelse ifølge krav 24, hvor kommunikationsdomænet hos mennesket forbedres.
27. Sammensætning til anvendelse ifølge krav 24, hvor den kognitive funktion hos mennesket forbedres.
28. Sammensætning til anvendelse ved reduktion af mindst ét element valgt fra gruppen bestående af irritabilitet og aggression hos et menneske med fragilt X-syndrom, hvor sammensætningen indbefatter en terapeutisk effektiv mængde af mindst én gamma-aminosmørsyre-agonist.
29. Sammensætning til anvendelse ved forbedring af mindst ét element valgt frz gruppen bestående af et socialt domæne og et kommunikationsdomæne hos et menneske med fragilt X-syndrom, hvor sammensætningen er en oral sammensætning, som indbefatter en terapeutisk effektiv mængde med mindst ca. 99 molprocent R-baclofen i forhold til det totale R-baclofen og S-baclofen.
30. Sammensætning til anvendelse ved reduktion af mindst ét element valgt fra gruppen bestående af irritabilitet og aggression hos et menneske med fragilt X-syndrom, hvor sammensætningen er en oral sammensætning, som indbefatter en terapeutisk effektiv mængde med mindst ca. 99 molprocent R-baclofen i forhold til det totale R-baclofen og S-baclofen.
DK07862184.4T 2006-11-22 2007-11-21 Fremgangsmåder til behandling af downs syndrom, fragilt x-syndrom og autisme DK2083811T3 (da)

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US86073306P 2006-11-22 2006-11-22
US156707P 2007-11-02 2007-11-02
PCT/US2007/024311 WO2008066750A1 (en) 2006-11-22 2007-11-21 Methods of treating mental retardation, down's syndrome, fragile x syndrome and autism

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