BR112018006445A2 - métodos para tratar distrofia muscular - Google Patents

métodos para tratar distrofia muscular

Info

Publication number
BR112018006445A2
BR112018006445A2 BR112018006445A BR112018006445A BR112018006445A2 BR 112018006445 A2 BR112018006445 A2 BR 112018006445A2 BR 112018006445 A BR112018006445 A BR 112018006445A BR 112018006445 A BR112018006445 A BR 112018006445A BR 112018006445 A2 BR112018006445 A2 BR 112018006445A2
Authority
BR
Brazil
Prior art keywords
muscular dystrophy
methods
treat muscular
disclosure provides
treating
Prior art date
Application number
BR112018006445A
Other languages
English (en)
Inventor
M Kaye Edward
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 BR112018006445A2 publication Critical patent/BR112018006445A2/pt

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)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Epidemiology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Neurology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Organic Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Orthopedic Medicine & Surgery (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

a presente divulgação fornece, entre outras coisas, composições e métodos melhorados para tratar distrofia muscular. por exemplo, a divulgação fornece métodos para tratar distrofia muscular de duchenne em pacientes tendo uma mutação no gene da dmd que é suscetível ao salto de éxon 51 administrando-se uma quantidade eficaz de eteplirsen.
BR112018006445A 2015-09-30 2016-09-29 métodos para tratar distrofia muscular BR112018006445A2 (pt)

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
BR112018006445A2 true BR112018006445A2 (pt) 2018-12-11

Family

ID=58424307

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112018006445A BR112018006445A2 (pt) 2015-09-30 2016-09-29 métodos para tratar distrofia muscular

Country Status (9)

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

Families Citing this family (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7807816B2 (en) 2004-06-28 2010-10-05 University Of Western Australia Antisense oligonucleotides for inducing exon skipping and methods of use thereof
HUE040445T2 (hu) 2009-11-12 2019-03-28 Univ Western Australia Antiszensz molekulák és eljárások betegségek kezelésére
TWI541024B (zh) 2010-09-01 2016-07-11 日本新藥股份有限公司 反義核酸
BR122020016865B1 (pt) 2013-03-14 2022-12-27 Sarepta Therapeutics, Inc. Oligonucleotídeo antisenso, composição farmacêutica compreendendo o mesmo e uso da dita composição para o tratamento de distrofia muscular de duchenne (dmd)
CA2906812A1 (en) 2013-03-15 2014-09-18 Sarepta Therapeutics, Inc. Improved compositions for treating muscular dystrophy
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
CN112399849A (zh) * 2018-06-26 2021-02-23 日本新药株式会社 含有反义寡核苷酸的组合物及其在迪谢内肌营养不良症的治疗中的用途
WO2023168427A1 (en) 2022-03-03 2023-09-07 Yale University Compositions and methods for delivering therapeutic polynucleotides for exon skipping
TW202342070A (zh) * 2022-03-30 2023-11-01 美商拜奧馬林製藥公司 肌萎縮蛋白外顯子跳躍寡核苷酸

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CA2906812A1 (en) * 2013-03-15 2014-09-18 Sarepta Therapeutics, Inc. Improved compositions for treating muscular dystrophy
KR102268473B1 (ko) * 2013-04-20 2021-06-25 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 엑손 2-표적 U7snRNA 폴리뉴클레오티드 작제물의 재조합형 아데노 부속 바이러스 전달

Also Published As

Publication number Publication date
WO2017059131A1 (en) 2017-04-06
JP2018529715A (ja) 2018-10-11
CN108738310A (zh) 2018-11-02
EP3355892A4 (en) 2019-05-22
EP3355892A1 (en) 2018-08-08
IL258211A (en) 2018-05-31
US20190054113A1 (en) 2019-02-21
US20210008095A1 (en) 2021-01-14
HK1258823A1 (zh) 2019-11-22
EP3653216A1 (en) 2020-05-20
EA201890811A1 (ru) 2018-09-28

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B07D Technical examination (opinion) related to article 229 of industrial property law [chapter 7.4 patent gazette]

Free format text: DE ACORDO COM O ARTIGO 229-C DA LEI NO 10196/2001, QUE MODIFICOU A LEI NO 9279/96, A CONCESSAO DA PATENTE ESTA CONDICIONADA A ANUENCIA PREVIA DA ANVISA. CONSIDERANDO A APROVACAO DOS TERMOS DO PARECER NO 337/PGF/EA/2010, BEM COMO A PORTARIA INTERMINISTERIAL NO 1065 DE 24/05/2012, ENCAMINHA-SE O PRESENTE PEDIDO PARA AS PROVIDENCIAS CABIVEIS.

B07E Notification of approval relating to section 229 industrial property law [chapter 7.5 patent gazette]
B07A Application suspended after technical examination (opinion) [chapter 7.1 patent gazette]
B09B Patent application refused [chapter 9.2 patent gazette]
B09B Patent application refused [chapter 9.2 patent gazette]

Free format text: MANTIDO O INDEFERIMENTO UMA VEZ QUE NAO FOI APRESENTADO RECURSO DENTRO DO PRAZO LEGAL

B350 Update of information on the portal [chapter 15.35 patent gazette]