TW202302156A - 用於治療x連鎖肌微管性肌病之組合物及方法 - Google Patents
用於治療x連鎖肌微管性肌病之組合物及方法 Download PDFInfo
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- TW202302156A TW202302156A TW111109009A TW111109009A TW202302156A TW 202302156 A TW202302156 A TW 202302156A TW 111109009 A TW111109009 A TW 111109009A TW 111109009 A TW111109009 A TW 111109009A TW 202302156 A TW202302156 A TW 202302156A
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- 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
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- 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
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- 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
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- 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
- 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
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- 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
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- 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
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- 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
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- 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
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Medicinal Chemistry (AREA)
- Biomedical Technology (AREA)
- Pharmacology & Pharmacy (AREA)
- Organic Chemistry (AREA)
- Genetics & Genomics (AREA)
- Molecular Biology (AREA)
- Anesthesiology (AREA)
- Epidemiology (AREA)
- Emergency Medicine (AREA)
- Pulmonology (AREA)
- Biotechnology (AREA)
- Heart & Thoracic Surgery (AREA)
- Hematology (AREA)
- Biochemistry (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Physical Education & Sports Medicine (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Neurology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Gastroenterology & Hepatology (AREA)
- Zoology (AREA)
- Toxicology (AREA)
- Biophysics (AREA)
- Proteomics, Peptides & Aminoacids (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)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202163159898P | 2021-03-11 | 2021-03-11 | |
| US63/159,898 | 2021-03-11 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| TW202302156A true TW202302156A (zh) | 2023-01-16 |
Family
ID=83227148
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| TW111109009A TW202302156A (zh) | 2021-03-11 | 2022-03-11 | 用於治療x連鎖肌微管性肌病之組合物及方法 |
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)
| 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 |
| 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 |
| JP2012531246A (ja) * | 2009-06-24 | 2012-12-10 | オリディオン メディカル 1987 リミテッド | 人工呼吸から離脱させる統合肺指標 |
| 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 |
| CN115052515B (zh) * | 2019-12-05 | 2026-01-30 | 迪萨蒂医疗公司 | 用于确定患者呼吸时所付出的呼吸努力程度的系统和方法 |
| WO2021142336A1 (en) * | 2020-01-08 | 2021-07-15 | Puretech Lyt, Inc. | Vesicle compositions for oral delivery |
| EP4351725A4 (en) * | 2021-05-24 | 2025-04-23 | Astellas Gene Therapies, Inc. | Compositions and methods for improved treatment of cross-linked myotube myopathy |
-
2022
- 2022-03-11 AR ARP220100570A patent/AR125077A1/es unknown
- 2022-03-11 US US18/281,346 patent/US20240148906A1/en active Pending
- 2022-03-11 EP EP22768049.3A patent/EP4304664A4/en active Pending
- 2022-03-11 TW TW111109009A patent/TW202302156A/zh unknown
- 2022-03-11 JP JP2023555441A patent/JP2024510972A/ja active Pending
- 2022-03-11 WO PCT/US2022/019876 patent/WO2022192622A1/en not_active Ceased
- 2022-03-11 CN CN202280029197.4A patent/CN117255698A/zh active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| JP2024510972A (ja) | 2024-03-12 |
| AR125077A1 (es) | 2023-06-07 |
| WO2022192622A1 (en) | 2022-09-15 |
| US20240148906A1 (en) | 2024-05-09 |
| EP4304664A4 (en) | 2025-07-30 |
| EP4304664A1 (en) | 2024-01-17 |
| CN117255698A (zh) | 2023-12-19 |
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