AR125077A1 - Composiciones y métodos para tratar la miopatía miotubular ligada a x - Google Patents

Composiciones y métodos para tratar la miopatía miotubular ligada a x

Info

Publication number
AR125077A1
AR125077A1 ARP220100570A ARP220100570A AR125077A1 AR 125077 A1 AR125077 A1 AR 125077A1 AR P220100570 A ARP220100570 A AR P220100570A AR P220100570 A ARP220100570 A AR P220100570A AR 125077 A1 AR125077 A1 AR 125077A1
Authority
AR
Argentina
Prior art keywords
approximately
mmhg
patient
cmh2o
respirator
Prior art date
Application number
ARP220100570A
Other languages
English (en)
Spanish (es)
Inventor
Geovanny Perez
Robert Graham
Salvador Rico
Suyash Prasad
Original Assignee
Audentes 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 Audentes Therapeutics Inc filed Critical Audentes Therapeutics Inc
Publication of AR125077A1 publication Critical patent/AR125077A1/es

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61M—DEVICES FOR INTRODUCING MEDIA INTO, OR ONTO, THE BODY; DEVICES FOR TRANSDUCING BODY MEDIA OR FOR TAKING MEDIA FROM THE BODY; DEVICES FOR PRODUCING OR ENDING SLEEP OR STUPOR
    • A61M16/00—Devices for influencing the respiratory system of patients by gas treatment, e.g. ventilators; Tracheal tubes
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61M—DEVICES FOR INTRODUCING MEDIA INTO, OR ONTO, THE BODY; DEVICES FOR TRANSDUCING BODY MEDIA OR FOR TAKING MEDIA FROM THE BODY; DEVICES FOR PRODUCING OR ENDING SLEEP OR STUPOR
    • A61M16/00—Devices for influencing the respiratory system of patients by gas treatment, e.g. ventilators; Tracheal tubes
    • A61M16/021—Devices for influencing the respiratory system of patients by gas treatment, e.g. ventilators; Tracheal tubes operated by electrical means
    • A61M16/022—Control means therefor
    • A61M16/024—Control means therefor including calculation means, e.g. using a processor
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4716—Muscle proteins, e.g. myosin, actin
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61M—DEVICES FOR INTRODUCING MEDIA INTO, OR ONTO, THE BODY; DEVICES FOR TRANSDUCING BODY MEDIA OR FOR TAKING MEDIA FROM THE BODY; DEVICES FOR PRODUCING OR ENDING SLEEP OR STUPOR
    • A61M16/00—Devices for influencing the respiratory system of patients by gas treatment, e.g. ventilators; Tracheal tubes
    • A61M16/0003—Accessories therefor, e.g. sensors, vibrators, negative pressure
    • A61M2016/0027—Accessories therefor, e.g. sensors, vibrators, negative pressure pressure meter
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61M—DEVICES FOR INTRODUCING MEDIA INTO, OR ONTO, THE BODY; DEVICES FOR TRANSDUCING BODY MEDIA OR FOR TAKING MEDIA FROM THE BODY; DEVICES FOR PRODUCING OR ENDING SLEEP OR STUPOR
    • A61M2205/00—General characteristics of the apparatus
    • A61M2205/33—Controlling, regulating or measuring
    • A61M2205/3306—Optical measuring means
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61M—DEVICES FOR INTRODUCING MEDIA INTO, OR ONTO, THE BODY; DEVICES FOR TRANSDUCING BODY MEDIA OR FOR TAKING MEDIA FROM THE BODY; DEVICES FOR PRODUCING OR ENDING SLEEP OR STUPOR
    • A61M2205/00—General characteristics of the apparatus
    • A61M2205/33—Controlling, regulating or measuring
    • A61M2205/3327—Measuring
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61M—DEVICES FOR INTRODUCING MEDIA INTO, OR ONTO, THE BODY; DEVICES FOR TRANSDUCING BODY MEDIA OR FOR TAKING MEDIA FROM THE BODY; DEVICES FOR PRODUCING OR ENDING SLEEP OR STUPOR
    • A61M2230/00—Measuring parameters of the user
    • A61M2230/20—Blood composition characteristics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61M—DEVICES FOR INTRODUCING MEDIA INTO, OR ONTO, THE BODY; DEVICES FOR TRANSDUCING BODY MEDIA OR FOR TAKING MEDIA FROM THE BODY; DEVICES FOR PRODUCING OR ENDING SLEEP OR STUPOR
    • A61M2230/00—Measuring parameters of the user
    • A61M2230/20—Blood composition characteristics
    • A61M2230/202—Blood composition characteristics partial carbon oxide pressure, e.g. partial dioxide pressure (P-CO2)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61M—DEVICES FOR INTRODUCING MEDIA INTO, OR ONTO, THE BODY; DEVICES FOR TRANSDUCING BODY MEDIA OR FOR TAKING MEDIA FROM THE BODY; DEVICES FOR PRODUCING OR ENDING SLEEP OR STUPOR
    • A61M2230/00—Measuring parameters of the user
    • A61M2230/20—Blood composition characteristics
    • A61M2230/205—Blood composition characteristics partial oxygen pressure (P-O2)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61M—DEVICES FOR INTRODUCING MEDIA INTO, OR ONTO, THE BODY; DEVICES FOR TRANSDUCING BODY MEDIA OR FOR TAKING MEDIA FROM THE BODY; DEVICES FOR PRODUCING OR ENDING SLEEP OR STUPOR
    • A61M2230/00—Measuring parameters of the user
    • A61M2230/40—Respiratory characteristics
    • A61M2230/42—Rate
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61M—DEVICES FOR INTRODUCING MEDIA INTO, OR ONTO, THE BODY; DEVICES FOR TRANSDUCING BODY MEDIA OR FOR TAKING MEDIA FROM THE BODY; DEVICES FOR PRODUCING OR ENDING SLEEP OR STUPOR
    • A61M2230/00—Measuring parameters of the user
    • A61M2230/40—Respiratory characteristics
    • A61M2230/43—Composition of exhalation
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61M—DEVICES FOR INTRODUCING MEDIA INTO, OR ONTO, THE BODY; DEVICES FOR TRANSDUCING BODY MEDIA OR FOR TAKING MEDIA FROM THE BODY; DEVICES FOR PRODUCING OR ENDING SLEEP OR STUPOR
    • A61M2230/00—Measuring parameters of the user
    • A61M2230/40—Respiratory characteristics
    • A61M2230/43—Composition of exhalation
    • A61M2230/432—Composition of exhalation partial CO2 pressure (P-CO2)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61M—DEVICES FOR INTRODUCING MEDIA INTO, OR ONTO, THE BODY; DEVICES FOR TRANSDUCING BODY MEDIA OR FOR TAKING MEDIA FROM THE BODY; DEVICES FOR PRODUCING OR ENDING SLEEP OR STUPOR
    • A61M2230/00—Measuring parameters of the user
    • A61M2230/63—Motion, e.g. physical activity
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biomedical Technology (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Emergency Medicine (AREA)
  • Pulmonology (AREA)
  • Anesthesiology (AREA)
  • Epidemiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Hematology (AREA)
  • Biochemistry (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Neurology (AREA)
  • Biophysics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Zoology (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines Containing Plant Substances (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
ARP220100570A 2021-03-11 2022-03-11 Composiciones y métodos para tratar la miopatía miotubular ligada a x AR125077A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US202163159898P 2021-03-11 2021-03-11

Publications (1)

Publication Number Publication Date
AR125077A1 true AR125077A1 (es) 2023-06-07

Family

ID=83227148

Family Applications (1)

Application Number Title Priority Date Filing Date
ARP220100570A AR125077A1 (es) 2021-03-11 2022-03-11 Composiciones y métodos para tratar la miopatía miotubular ligada a x

Country Status (7)

Country Link
US (1) US20240148906A1 (https=)
EP (1) EP4304664A4 (https=)
JP (1) JP2024510972A (https=)
CN (1) CN117255698A (https=)
AR (1) AR125077A1 (https=)
TW (1) TW202302156A (https=)
WO (1) WO2022192622A1 (https=)

Family Cites Families (23)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SE9502543D0 (sv) * 1995-07-10 1995-07-10 Lachmann Burkhardt Artificial ventilation system
US6148814A (en) * 1996-02-08 2000-11-21 Ihc Health Services, Inc Method and system for patient monitoring and respiratory assistance control through mechanical ventilation by the use of deterministic protocols
US6804656B1 (en) * 1999-06-23 2004-10-12 Visicu, Inc. System and method for providing continuous, expert network critical care services from a remote location(s)
US7246618B2 (en) * 2001-06-21 2007-07-24 Nader Maher Habashi Ventilation method and control of a ventilator based on same
WO2010150264A1 (en) * 2009-06-24 2010-12-29 Oridion Medical 1987 Ltd. Integrated pulmonary index for weaning from mechanical ventilation
US8695593B2 (en) * 2007-03-31 2014-04-15 Fleur T. Tehrani Weaning and decision support system for mechanical ventilation
US20080295839A1 (en) * 2007-06-01 2008-12-04 Habashi Nader M Ventilator Apparatus and System of Ventilation
US8789529B2 (en) * 2009-08-20 2014-07-29 Covidien Lp Method for ventilation
US20160199608A1 (en) * 2010-02-18 2016-07-14 Orlando Morejon System and method of expediting weaning from ventilator support including an artificial airway cleaning apparatus
US20110213215A1 (en) * 2010-02-26 2011-09-01 Nellcor Puritan Bennett Llc Spontaneous Breathing Trial Manager
US20180280646A1 (en) * 2010-08-13 2018-10-04 Respiratory Motion, Inc. Respiratory volume monitor and ventilator
US9327090B2 (en) * 2012-06-29 2016-05-03 Carefusion 303, Inc. Respiratory knowledge portal
US20140060540A1 (en) * 2012-09-06 2014-03-06 Nellcor Puritan Bennett Llc Method and system for automated spontaneous breathing trial
US20140150796A1 (en) * 2012-11-30 2014-06-05 Covidien Lp System and method for detecting minimal ventilation support with proportional assist ventilation plus software and remote monitoring
US20140235959A1 (en) * 2013-02-15 2014-08-21 Covidien Lp Methods and algorithms for supervisory closed-loop determination of optimized scheduling of ventilator weaning trials
US8957044B2 (en) * 2013-03-01 2015-02-17 Wake Forest University Health Sciences Systemic gene replacement therapy for treatment of X-linked myotubular myopathy (XLMTM)
WO2018035311A1 (en) * 2016-08-19 2018-02-22 Gray John T Compositions and methods for modulating gene expression using reading frame surveillance
FI128346B (en) * 2018-11-19 2020-03-31 Hapella Oy A device for taking care of respiratory well-being and for practicing and improving respiratory function
US11166862B2 (en) * 2018-12-06 2021-11-09 General Electric Company System and method for a thermoregulated environment
WO2021110576A1 (en) * 2019-12-02 2021-06-10 Koninklijke Philips N.V. Providing information for assisting in the treatment of a ventilated and sedated patient
AU2020397171A1 (en) * 2019-12-05 2022-07-07 Disati Medical, Inc Systems and methods for determining a degree of respiratory effort exerted by a patient while breathing
US20230157955A1 (en) * 2020-01-08 2023-05-25 Puretech Lyt, Inc. Vesicle compositions for oral delivery
PH12023553216A1 (en) * 2021-05-24 2024-04-29 Astellas Gene Therapies Inc Compositions and methods for improved treatment of x-linked myotubular myopathy

Also Published As

Publication number Publication date
WO2022192622A1 (en) 2022-09-15
TW202302156A (zh) 2023-01-16
US20240148906A1 (en) 2024-05-09
JP2024510972A (ja) 2024-03-12
CN117255698A (zh) 2023-12-19
EP4304664A1 (en) 2024-01-17
EP4304664A4 (en) 2025-07-30

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