EA201890811A1 - Способы лечения мышечной дистрофии - Google Patents

Способы лечения мышечной дистрофии

Info

Publication number
EA201890811A1
EA201890811A1 EA201890811A EA201890811A EA201890811A1 EA 201890811 A1 EA201890811 A1 EA 201890811A1 EA 201890811 A EA201890811 A EA 201890811A EA 201890811 A EA201890811 A EA 201890811A EA 201890811 A1 EA201890811 A1 EA 201890811A1
Authority
EA
Eurasian Patent Office
Prior art keywords
methods
distrophia
muscular
treatment
muscular dystrophy
Prior art date
Application number
EA201890811A
Other languages
English (en)
Inventor
Эдвард М. Кайе
Original Assignee
Сарепта Терапьютикс, Инк.
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 Сарепта Терапьютикс, Инк. filed Critical Сарепта Терапьютикс, Инк.
Publication of EA201890811A1 publication Critical patent/EA201890811A1/ru

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
    • 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
    • A61K31/56—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids
    • A61K31/57—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids substituted in position 17 beta by a chain of two carbon atoms, e.g. pregnane or progesterone
    • A61K31/573—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids substituted in position 17 beta by a chain of two carbon atoms, e.g. pregnane or progesterone substituted in position 21, e.g. cortisone, dexamethasone, prednisone or aldosterone
    • 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
    • A61K31/56—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids
    • A61K31/58—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids containing heterocyclic rings, e.g. danazol, stanozolol, pancuronium or digitogenin
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • 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
    • 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
    • A61P21/06—Anabolic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Neurology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Endocrinology (AREA)
  • Dermatology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)

Abstract

Настоящее раскрытие, среди прочего, обеспечивает усовершенствованные композиции и способы лечения мышечной дистрофии. Например, раскрытие обеспечивает способы лечения пациентов с мышечной дистрофией Дюшенна, имеющих мутацию в гене DMD, поддающуюся корректировке пропуском экзона 51, введением эффективного количества этеплирсена.
EA201890811A 2015-09-30 2016-09-29 Способы лечения мышечной дистрофии EA201890811A1 (ru)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201562235477P 2015-09-30 2015-09-30
US201662278866P 2016-01-14 2016-01-14
US201662293235P 2016-02-09 2016-02-09
US201662299952P 2016-02-25 2016-02-25
PCT/US2016/054534 WO2017059131A1 (en) 2015-09-30 2016-09-29 Methods for treating muscular dystrophy

Publications (1)

Publication Number Publication Date
EA201890811A1 true EA201890811A1 (ru) 2018-09-28

Family

ID=58424307

Family Applications (1)

Application Number Title Priority Date Filing Date
EA201890811A EA201890811A1 (ru) 2015-09-30 2016-09-29 Способы лечения мышечной дистрофии

Country Status (9)

Country Link
US (2) US20190054113A1 (ru)
EP (2) EP3653216A1 (ru)
JP (1) JP2018529715A (ru)
CN (1) CN108738310A (ru)
BR (1) BR112018006445A2 (ru)
EA (1) EA201890811A1 (ru)
HK (1) HK1258823A1 (ru)
IL (1) IL258211A (ru)
WO (1) WO2017059131A1 (ru)

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Publication number Priority date Publication date Assignee Title
ES2361325T3 (es) 2004-06-28 2011-06-16 The University Of Western Australia Oligonucleótidos antisentido para inducir la omisión de exón y métodos de uso de los mismos.
EP3431603A1 (en) 2009-11-12 2019-01-23 The University Of Western Australia Antisense molecules and methods for treating pathologies
TWI541024B (zh) 2010-09-01 2016-07-11 日本新藥股份有限公司 反義核酸
IL280443B (en) 2013-03-14 2022-07-01 Sarepta Therapeutics Inc Preparations that skip axon for the treatment of muscular dystrophy
EA201591792A1 (ru) 2013-03-15 2016-02-29 Сарепта Терапьютикс, Инк. Улучшенные композиции для лечения мышечной дистрофии
US11083737B1 (en) * 2016-05-27 2021-08-10 Ptc Therapeutics, Inc. Methods of administering deflazacort therapy
GB201711809D0 (en) 2017-07-21 2017-09-06 Governors Of The Univ Of Alberta Antisense oligonucleotide
MX2020013880A (es) * 2018-06-26 2021-03-09 Nippon Shinyaku Co Ltd Composición que comprende el oligonucleótido antisentido y su uso para el tratamiento de la distrofia muscular de duchenne.
JP2022526763A (ja) * 2019-03-28 2022-05-26 サレプタ セラピューティクス， インコーポレイテッド カシメルセンによる筋ジストロフィーの治療方法
WO2023168427A1 (en) 2022-03-03 2023-09-07 Yale University Compositions and methods for delivering therapeutic polynucleotides for exon skipping
US20230348909A1 (en) * 2022-03-30 2023-11-02 Biomarin Pharmaceutical Inc. Dystrophin exon skipping oligonucleotides

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CA2909807C (en) * 2013-04-20 2023-08-08 Research Institute At Nationwide Children's Hospital Recombinant adeno-associated virus delivery of exon 2-targeted u7snrna polynucleotide constructs

Also Published As

Publication number Publication date
EP3653216A1 (en) 2020-05-20
US20210008095A1 (en) 2021-01-14
HK1258823A1 (zh) 2019-11-22
BR112018006445A2 (pt) 2018-12-11
US20190054113A1 (en) 2019-02-21
WO2017059131A1 (en) 2017-04-06
EP3355892A1 (en) 2018-08-08
CN108738310A (zh) 2018-11-02
JP2018529715A (ja) 2018-10-11
IL258211A (en) 2018-05-31
EP3355892A4 (en) 2019-05-22

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