JP2022553888A - レット症候群の治療のための方法及び組成物 - Google Patents

レット症候群の治療のための方法及び組成物 Download PDF

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JP2022553888A
JP2022553888A JP2022552123A JP2022552123A JP2022553888A JP 2022553888 A JP2022553888 A JP 2022553888A JP 2022552123 A JP2022552123 A JP 2022552123A JP 2022552123 A JP2022552123 A JP 2022552123A JP 2022553888 A JP2022553888 A JP 2022553888A
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trophinetide
daily dose
administered
use according
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JP2022553888A5 (https=
Inventor
モナ ダーウィッシュ，
ジェームス エム． ヨウアキム，
ローレンス アーウィン グラス，
ナンシー エリザベス ジョーンズ，
ショーン ポール オースターホルト，
オスカー デラ パスクア，
Original Assignee
アカディア ファーマシューティカルズ インコーポレーテッド
ニューレン ファーマシューティカルズ リミテッド
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Publication of JP2022553888A publication Critical patent/JP2022553888A/ja
Publication of JP2022553888A5 publication Critical patent/JP2022553888A5/ja
Priority to JP2025116586A priority Critical patent/JP2025148461A/ja
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/04—Peptides having up to 20 amino acids in a fully defined sequence; Derivatives thereof
    • A61K38/06—Tripeptides
    • 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
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/40—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with one nitrogen as the only ring hetero atom, e.g. sulpiride, succinimide, tolmetin, buflomedil
    • 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
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/40—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with one nitrogen as the only ring hetero atom, e.g. sulpiride, succinimide, tolmetin, buflomedil
    • A61K31/401—Proline; Derivatives thereof, e.g. captopril
    • 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
    • A61K9/0053—Mouth and digestive tract, i.e. intraoral and peroral administration
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K5/00—Peptides containing up to four amino acids in a fully defined sequence; Derivatives thereof
    • C07K5/04—Peptides containing up to four amino acids in a fully defined sequence; Derivatives thereof containing only normal peptide links
    • C07K5/08—Tripeptides

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Epidemiology (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Psychiatry (AREA)
  • Physiology (AREA)
  • Nutrition Science (AREA)
  • Hospice & Palliative Care (AREA)
  • Biochemistry (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Detergent Compositions (AREA)
  • Chemical Or Physical Treatment Of Fibers (AREA)
  • Peptides Or Proteins (AREA)
JP2022552123A 2019-10-28 2020-10-28 レット症候群の治療のための方法及び組成物 Pending JP2022553888A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2025116586A JP2025148461A (ja) 2019-10-28 2025-07-10 レット症候群の治療のための方法及び組成物

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201962927008P 2019-10-28 2019-10-28
US62/927,008 2019-10-28
US202063031201P 2020-05-28 2020-05-28
US63/031,201 2020-05-28
PCT/US2020/057627 WO2021086892A1 (en) 2019-10-28 2020-10-28 Methods and compositions for treatment of rett syndrome

Related Child Applications (1)

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JP2025116586A Division JP2025148461A (ja) 2019-10-28 2025-07-10 レット症候群の治療のための方法及び組成物

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JP2022553888A true JP2022553888A (ja) 2022-12-26
JP2022553888A5 JP2022553888A5 (https=) 2023-10-18

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JP2022552123A Pending JP2022553888A (ja) 2019-10-28 2020-10-28 レット症候群の治療のための方法及び組成物
JP2025116586A Pending JP2025148461A (ja) 2019-10-28 2025-07-10 レット症候群の治療のための方法及び組成物

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Country Link
US (1) US20220339138A1 (https=)
EP (1) EP4051308A4 (https=)
JP (2) JP2022553888A (https=)
KR (1) KR20220106982A (https=)
CN (1) CN115335071A (https=)
AU (1) AU2020376801A1 (https=)
BR (1) BR112022008095A2 (https=)
CA (1) CA3156680A1 (https=)
CL (1) CL2022001079A1 (https=)
CO (1) CO2022007501A2 (https=)
IL (1) IL292617B2 (https=)
MX (1) MX2022004785A (https=)
TW (1) TW202116300A (https=)
WO (1) WO2021086892A1 (https=)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
IL310045A (en) 2021-07-12 2024-03-01 Acadia Pharm Inc Crystalline forms of tropintide
WO2023242844A1 (en) * 2022-06-15 2023-12-21 Ramot At Tel-Aviv University Ltd. Method and therapeutic agent for treatment of disease or disorder associated with impaired firing rate and/or mitochondrial calcium homeostasis
WO2025262646A1 (en) * 2024-06-22 2025-12-26 Biophore India Pharmaceuticals Pvt. Ltd Dosage forms of trofinetide and its pharmaceutically acceptable salt thereof

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6709817B1 (en) * 1999-09-07 2004-03-23 Baylor College Of Medicine Method of screening Rett syndrome by detecting a mutation in MECP2
US9708366B2 (en) * 2011-01-27 2017-07-18 Neuren Pharmaceuticals Ltd. Treatment of fragile X syndrome using glycyl-L-2-methylprolyl-L-glutamate
WO2014130691A2 (en) * 2013-02-20 2014-08-28 Theravasc Inc. Pharmaceutical formulations of nitrite and uses thereof
WO2015013397A2 (en) * 2013-07-25 2015-01-29 Neuren Pharmaceuticals Limited Neuroprotective bicyclic compounds and methods for their use in treating autism spectrum disorders and neurodevelopmental disorders
US20150224164A1 (en) * 2013-11-26 2015-08-13 Neuren Pharmaceuticals Limited Treatment of autism spectrum disorders using glycyl-l-2-methylprolyl-l-glumatic acid
GB2554064B (en) * 2016-09-01 2019-11-20 Jit Singh Shailinder An enteral feeding tube

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
NEUROLOGY, vol. Volume 92, Number 16, JPN6024038594, 16 April 2019 (2019-04-16), pages 1912 - 1925, ISSN: 0005701227 *

Also Published As

Publication number Publication date
CN115335071A (zh) 2022-11-11
TW202116300A (zh) 2021-05-01
CA3156680A1 (en) 2021-05-06
CL2022001079A1 (es) 2023-04-21
EP4051308A4 (en) 2023-08-23
MX2022004785A (es) 2022-05-16
CO2022007501A2 (es) 2022-08-30
KR20220106982A (ko) 2022-08-01
US20220339138A1 (en) 2022-10-27
AU2020376801A1 (en) 2022-06-09
IL292617B1 (en) 2025-11-01
IL292617B2 (en) 2026-03-01
EP4051308A1 (en) 2022-09-07
WO2021086892A1 (en) 2021-05-06
BR112022008095A2 (pt) 2022-07-12
IL292617A (en) 2022-07-01
JP2025148461A (ja) 2025-10-07

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