AR112538A1 - Métodos para el tratamiento de distrofia muscular - Google Patents
Métodos para el tratamiento de distrofia muscularInfo
- 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
Links
Classifications
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- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Biomedical Technology (AREA)
- Medicinal Chemistry (AREA)
- Molecular Biology (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Genetics & Genomics (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Biochemistry (AREA)
- Epidemiology (AREA)
- Biotechnology (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Physics & Mathematics (AREA)
- Neurology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Physical Education & Sports Medicine (AREA)
- Microbiology (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Immunology (AREA)
- Urology & Nephrology (AREA)
- Hematology (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Cell Biology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Food Science & Technology (AREA)
- Analytical Chemistry (AREA)
- General Physics & Mathematics (AREA)
- Pathology (AREA)
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.
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 (5)
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 |
EP3987029A1 (en) * | 2019-06-19 | 2022-04-27 | Sarepta Therapeutics, Inc. | Methods for treating muscular dystrophy |
US20230256047A1 (en) * | 2020-06-17 | 2023-08-17 | Stealth Biotherapeutics Inc. | Methods and compositions for the treatment of 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)
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 |
MX366274B (es) * | 2013-03-14 | 2019-07-04 | Sarepta Therapeutics Inc | Composiciones para el salto del exón para el tratamiento de distrofia muscular. |
BR112015022998A2 (pt) * | 2013-03-15 | 2017-11-14 | Sarepta Therapeutics Inc | composições melhoradas para o tratamento de distrofia muscular |
EP3359668A4 (en) * | 2015-10-09 | 2019-06-05 | Sarepta Therapeutics, Inc. | COMPOSITIONS AND METHODS OF TREATING DUCHENNE MUSCLE DYSTROPHY AND ASSOCIATED ILLNESSES THEREOF |
-
2018
- 2018-08-31 MX MX2020002038A patent/MX2020002038A/es unknown
- 2018-08-31 EP EP18851503.5A patent/EP3675836A4/en active Pending
- 2018-08-31 AR ARP180102486 patent/AR112538A1/es unknown
- 2018-08-31 AU AU2018326780A patent/AU2018326780A1/en active Pending
- 2018-08-31 US US16/640,852 patent/US20230038956A1/en not_active Abandoned
- 2018-08-31 JP JP2020511299A patent/JP2020532504A/ja not_active Withdrawn
- 2018-08-31 CN CN201880056909.5A patent/CN111417388A/zh active Pending
- 2018-08-31 CA CA3073736A patent/CA3073736A1/en active Pending
- 2018-08-31 WO PCT/US2018/049151 patent/WO2019046755A1/en unknown
- 2018-08-31 SG SG11202001074RA patent/SG11202001074RA/en unknown
- 2018-08-31 KR KR1020207008557A patent/KR20200046069A/ko not_active Application Discontinuation
- 2018-08-31 MA MA050062A patent/MA50062A/fr unknown
- 2018-08-31 TW TW107130738A patent/TW201919655A/zh unknown
-
2020
- 2020-02-20 IL IL272791A patent/IL272791A/en unknown
- 2020-03-31 CO CONC2020/0004034A patent/CO2020004034A2/es unknown
-
2023
- 2023-08-14 JP JP2023131995A patent/JP2023138867A/ja active Pending
Also Published As
Publication number | Publication date |
---|---|
US20230038956A1 (en) | 2023-02-09 |
CA3073736A1 (en) | 2019-03-07 |
SG11202001074RA (en) | 2020-03-30 |
CN111417388A (zh) | 2020-07-14 |
JP2023138867A (ja) | 2023-10-02 |
TW201919655A (zh) | 2019-06-01 |
EP3675836A1 (en) | 2020-07-08 |
EP3675836A4 (en) | 2021-05-26 |
AU2018326780A1 (en) | 2020-02-27 |
CO2020004034A2 (es) | 2020-04-24 |
KR20200046069A (ko) | 2020-05-06 |
MA50062A (fr) | 2020-07-08 |
IL272791A (en) | 2020-04-30 |
JP2020532504A (ja) | 2020-11-12 |
MX2020002038A (es) | 2020-09-18 |
WO2019046755A1 (en) | 2019-03-07 |
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