MX373959B - Composición para usarse en el tratamiento de la distrofia muscular de duchenne (dmd). - Google Patents

Composición para usarse en el tratamiento de la distrofia muscular de duchenne (dmd).

Info

Publication number
MX373959B
MX373959B MX2015013117A MX2015013117A MX373959B MX 373959 B MX373959 B MX 373959B MX 2015013117 A MX2015013117 A MX 2015013117A MX 2015013117 A MX2015013117 A MX 2015013117A MX 373959 B MX373959 B MX 373959B
Authority
MX
Mexico
Prior art keywords
muscular dystrophy
improved compositions
treating muscular
treating
eteplirsen
Prior art date
Application number
MX2015013117A
Other languages
English (en)
Other versions
MX2015013117A (es
Inventor
Edward M Kaye
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 MX2015013117A publication Critical patent/MX2015013117A/es
Publication of MX373959B publication Critical patent/MX373959B/es

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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
    • 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
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive 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
    • 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
    • A61P21/02—Muscle relaxants, e.g. for tetanus or cramps
    • 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
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • 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
    • C12N2310/33—Chemical structure of the base
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Epidemiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Neurology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Pain & Pain Management (AREA)
  • Dermatology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

Abstract

La presente invención se refiere a el uso de eteplirsen para la elaboración de un medicamento para el tratamiento de la distrofia muscular de duchenne (DMD) en un paciente quien tiene una mutación del gen de DMD que es responsable del salto del exón 51, en donde el medicamento está adaptado para ser administrable por vía intravenosa al paciente en una dosis de aproximadamente 30 mg/kg una vez por semana durante más de 120 semanas, para que se retrase en el paciente la progresión de la enfermedad.
MX2015013117A 2013-03-15 2014-03-14 Composición para usarse en el tratamiento de la distrofia muscular de duchenne (dmd). MX373959B (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201361793463P 2013-03-15 2013-03-15
PCT/US2014/029610 WO2014144978A2 (en) 2013-03-15 2014-03-14 Improved compositions for treating muscular dystrophy

Publications (2)

Publication Number Publication Date
MX2015013117A MX2015013117A (es) 2016-07-14
MX373959B true MX373959B (es) 2020-07-13

Family

ID=50678303

Family Applications (2)

Application Number Title Priority Date Filing Date
MX2015013117A MX373959B (es) 2013-03-15 2014-03-14 Composición para usarse en el tratamiento de la distrofia muscular de duchenne (dmd).
MX2020004986A MX2020004986A (es) 2013-03-15 2015-09-15 Composiciones mejoradas para tratar distrofia muscular.

Family Applications After (1)

Application Number Title Priority Date Filing Date
MX2020004986A MX2020004986A (es) 2013-03-15 2015-09-15 Composiciones mejoradas para tratar distrofia muscular.

Country Status (15)

Country Link
US (10) US20140329762A1 (es)
EP (2) EP2968991A2 (es)
JP (2) JP2016516066A (es)
KR (2) KR20200139271A (es)
CN (2) CN113633787A (es)
AU (3) AU2014233456B2 (es)
BR (1) BR112015022998A2 (es)
CA (1) CA2906812A1 (es)
EA (1) EA201591792A1 (es)
ES (1) ES3063113T3 (es)
HK (1) HK1220154A1 (es)
IL (3) IL241558B (es)
MX (2) MX373959B (es)
NZ (2) NZ732507A (es)
WO (1) WO2014144978A2 (es)

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