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1. WO2008045566 - METHODS FOR DOSING AN ORALLY ACTIVE 1,2,4-OXADIAZOLE FOR NONSENSE MUTATION SUPPRESSION THERAPY

Publication Number WO/2008/045566
Publication Date 17.04.2008
International Application No. PCT/US2007/021921
International Filing Date 11.10.2007
IPC
A61K 31/4245 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31Medicinal preparations containing organic active ingredients
33Heterocyclic compounds
395having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
41having five-membered rings with two or more ring hetero atoms, at least one of which is nitrogen, e.g. tetrazole
4245Oxadiazoles
A61P 3/10 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
3Drugs for disorders of the metabolism
08for glucose homeostasis
10for hyperglycaemia, e.g. antidiabetics
A61P 7/00 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
7Drugs for disorders of the blood or the extracellular fluid
A61P 9/00 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
9Drugs for disorders of the cardiovascular system
CPC
A61K 31/4245
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31Medicinal preparations containing organic active ingredients
33Heterocyclic compounds
395having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
41having five-membered rings with two or more ring hetero atoms, at least one of which being nitrogen, e.g. tetrazole
4245Oxadiazoles
A61K 47/02
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
47Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
02Inorganic compounds
A61K 47/10
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
47Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
06Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
08containing oxygen, ; e.g. ethers, acetals, ketones, quinones, aldehydes, peroxides
10Alcohols; Phenols; Salts thereof, e.g. glycerol; Polyethylene glycols [PEG]; Poloxamers; PEG/POE alkyl ethers
A61K 47/12
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
47Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
06Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
08containing oxygen, ; e.g. ethers, acetals, ketones, quinones, aldehydes, peroxides
12Carboxylic acids; Salts or anhydrides thereof
A61K 47/26
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
47Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
06Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
26Carbohydrates, e.g. sugar alcohols, amino sugars, nucleic acids, mono-, di- or oligo-saccharides; Derivatives thereof, e.g. polysorbates, sorbitan fatty acid esters or glycyrrhizin
A61K 47/30
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
47Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
30Macromolecular organic or inorganic compounds, e.g. inorganic polyphosphates
Applicants
  • PTC THERAPEUTICS, INC. [US]/[US] (AllExceptUS)
  • HIRAWAT, Samit [IN]/[US] (UsOnly)
  • MILLER, Langdon [US]/[US] (UsOnly)
Inventors
  • HIRAWAT, Samit
  • MILLER, Langdon
Agents
  • CORUZZI, Laura, A.
Priority Data
60/851,45012.10.2006US
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) METHODS FOR DOSING AN ORALLY ACTIVE 1,2,4-OXADIAZOLE FOR NONSENSE MUTATION SUPPRESSION THERAPY
(FR) PROCÉDÉS POUR DOSER LE 1,2,4-OXADIAOZLE ACTIF PAR VOIE ORALE POUR LA THÉRAPIE DE SUPPRESSION DES MUTATIONS NON-SENS
Abstract
(EN)
The present invention relates to specific doses of and dosing regimens for using a 1,2,4-oxadiazole benzoic acid compound in treating or preventing diseases associated with nonsense mutations. In particular, the invention relates to specific doses and dosing regimens for the use of 3-[5-(2-fluoro-phenyl)-[1,2,4]oxadiazol-3-yl]-benzoic acid in mammals having diseases associated with nonsense mutations.
(FR)
L'invention se rapporte à des doses et à des posologies spécifiques pour l'utilisation d'un composé qui est l'acide 1,2,4-oxadiazole benzoïque, pour le traitement ou la prévention de maladies associées à des mutations non-sens. L'invention a trait, en particulier, à des doses et à des posologies spécifiques destinées à l'utilisation de l'acide 3-[5-(2-fluoro-phényl)-[1,2,4]oxadiazol-3-yl]-benzoïque chez des mammifères atteints de maladies associées à des mutations non-sens.
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