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1. WO2020186217 - METHODS AND COMPOSITIONS FOR TREATMENT OF LYSOSOMAL STORAGE DISORDER

Publication Number WO/2020/186217
Publication Date 17.09.2020
International Application No. PCT/US2020/022761
International Filing Date 13.03.2020
IPC
A61K 35/761 2015.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
35Medicinal preparations containing materials or reaction products thereof with undetermined constitution
66Microorganisms or materials therefrom
76Viruses; Subviral particles; Bacteriophages
761Adenovirus
A61K 38/46 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
38Medicinal preparations containing peptides
16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
43Enzymes; Proenzymes; Derivatives thereof
46Hydrolases (3)
A61K 47/26 2006.01
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
Applicants
  • CHILDREN'S HOSPITAL MEDICAL CENTER [US]/[US]
Inventors
  • PAN, Dao
Agents
  • CHEN, Yahua
Priority Data
62/818,22014.03.2019US
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) METHODS AND COMPOSITIONS FOR TREATMENT OF LYSOSOMAL STORAGE DISORDER
(FR) MÉTHODES ET COMPOSITIONS DE TRAITEMENT DE TROUBLES D'ENTREPOSAGE LYSOSOMIAL
Abstract
(EN)
Methods for improving at least one neurological function in a subject that has or is suspected of having a neurologic lysosomal storage disorder using a rapamy cin compound. Also provided herein are treatment of such a neurologic lysosomal storage disorder with the rapamy cin compound.
(FR)
L'invention concerne des méthodes qui permettent d'améliorer au moins une fonction neurologique chez le patient qui est atteint, ou est suspecté d'être atteint, d'un trouble d'entreposage lysosomial de type neurologique à l'aide d'un composé de rapamycine. L'invention concerne également le traitement d'un tel trouble d'entreposage lysosomial de type neurologique avec le composé de rapamycine.
Latest bibliographic data on file with the International Bureau