AR112538A1 - Métodos para el tratamiento de distrofia muscular - Google Patents

Métodos para el tratamiento de distrofia muscular

Info

Publication number
AR112538A1
AR112538A1 ARP180102486A AR112538A1 AR 112538 A1 AR112538 A1 AR 112538A1 AR P180102486 A ARP180102486 A AR P180102486A AR 112538 A1 AR112538 A1 AR 112538A1
Authority
AR
Argentina
Prior art keywords
muscular dystrophy
methods
treatment
disclosure provides
treating
Prior art date
Application number
Other languages
English (en)
Original Assignee
Sarepta Therapeutics Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Sarepta Therapeutics Inc filed Critical Sarepta Therapeutics Inc
Publication of AR112538A1 publication Critical patent/AR112538A1/es

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/535—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with at least one nitrogen and one oxygen as the ring hetero atoms, e.g. 1,2-oxazines
    • A61K31/5375—1,4-Oxazines, e.g. morpholine
    • A61K31/5377—1,4-Oxazines, e.g. morpholine not condensed and containing further heterocyclic rings, e.g. timolol
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/68—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3233—Morpholino-type ring
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

Abstract

La presente descripción proporciona, entre otras cosas, composiciones y métodos mejorados para tratar la distrofia muscular. Por ejemplo, la descripción proporciona métodos para tratar pacientes con distrofia muscular de Duchenne que tienen una mutación en el gen DMD que es susceptible de omisión del exón 53 administrando una cantidad eficaz de golodirsen.
ARP180102486 2017-08-31 2018-08-31 Métodos para el tratamiento de distrofia muscular AR112538A1 (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762553094P 2017-08-31 2017-08-31
US201762565824P 2017-09-29 2017-09-29
US201862725129P 2018-08-30 2018-08-30

Publications (1)

Publication Number Publication Date
AR112538A1 true AR112538A1 (es) 2019-11-06

Family

ID=65526062

Family Applications (1)

Application Number Title Priority Date Filing Date
ARP180102486 AR112538A1 (es) 2017-08-31 2018-08-31 Métodos para el tratamiento de distrofia muscular

Country Status (15)

Country Link
US (1) US20230038956A1 (es)
EP (1) EP3675836A4 (es)
JP (2) JP2020532504A (es)
KR (1) KR20200046069A (es)
CN (1) CN111417388A (es)
AR (1) AR112538A1 (es)
AU (1) AU2018326780A1 (es)
CA (1) CA3073736A1 (es)
CO (1) CO2020004034A2 (es)
IL (1) IL272791A (es)
MA (1) MA50062A (es)
MX (1) MX2020002038A (es)
SG (1) SG11202001074RA (es)
TW (1) TW201919655A (es)
WO (1) WO2019046755A1 (es)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
TWI541024B (zh) 2010-09-01 2016-07-11 日本新藥股份有限公司 反義核酸
US20220296633A1 (en) * 2019-06-19 2022-09-22 Sarepta Therapeutics, Inc. Methods for treating muscular dystrophy
CN112430645A (zh) * 2020-12-09 2021-03-02 北京华瑞康源生物科技发展有限公司 一种多重实时荧光pcr法检测人dmd基因拷贝数的相对定量方法及试剂盒
WO2023168427A1 (en) 2022-03-03 2023-09-07 Yale University Compositions and methods for delivering therapeutic polynucleotides for exon skipping

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
TWI541024B (zh) * 2010-09-01 2016-07-11 日本新藥股份有限公司 反義核酸
HUE042218T2 (hu) * 2013-03-14 2019-06-28 Sarepta Therapeutics Inc Exonátugrást elõsegítõ készítmények izomdisztrófia kezelésére
BR112015022998A2 (pt) * 2013-03-15 2017-11-14 Sarepta Therapeutics Inc composições melhoradas para o tratamento de distrofia muscular
BR112018007066A2 (pt) * 2015-10-09 2018-10-23 Sarepta Therapeutics Inc composições e métodos para tratamento da distrofia muscular de duchene e distúrbios relacionados

Also Published As

Publication number Publication date
MX2020002038A (es) 2020-09-18
CO2020004034A2 (es) 2020-04-24
JP2023138867A (ja) 2023-10-02
CA3073736A1 (en) 2019-03-07
TW201919655A (zh) 2019-06-01
EP3675836A4 (en) 2021-05-26
MA50062A (fr) 2020-07-08
SG11202001074RA (en) 2020-03-30
AU2018326780A1 (en) 2020-02-27
IL272791A (en) 2020-04-30
WO2019046755A1 (en) 2019-03-07
EP3675836A1 (en) 2020-07-08
KR20200046069A (ko) 2020-05-06
JP2020532504A (ja) 2020-11-12
US20230038956A1 (en) 2023-02-09
CN111417388A (zh) 2020-07-14

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