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CL2022001079A1 - Métodos y composiciones para el tratamiento del síndrome de rett - Google Patents

Métodos y composiciones para el tratamiento del síndrome de rett

Info

Publication number
CL2022001079A1
CL2022001079A1 CL2022001079A CL2022001079A CL2022001079A1 CL 2022001079 A1 CL2022001079 A1 CL 2022001079A1 CL 2022001079 A CL2022001079 A CL 2022001079A CL 2022001079 A CL2022001079 A CL 2022001079A CL 2022001079 A1 CL2022001079 A1 CL 2022001079A1
Authority
CL
Chile
Prior art keywords
methods
rett syndrome
compositions
treatment
trofinetide
Prior art date
Application number
CL2022001079A
Other languages
English (en)
Inventor
Lawrence Irwin Glass
Mon Darwish
James M Youakim
Nancy Elizabeth Jones
Sean Paul Oosterholt
Oscar Della Pasqua
Original Assignee
Neuren Pharmaceuticals Ltd
Acadia Pharm 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 Neuren Pharmaceuticals Ltd, Acadia Pharm Inc filed Critical Neuren Pharmaceuticals Ltd
Publication of CL2022001079A1 publication Critical patent/CL2022001079A1/es

Links

Classifications

    • 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

Landscapes

  • 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)

Abstract

En la presente se divulgan métodos de tratamiento del síndrome de Rett que comprenden administrar trofinetide a un sujeto que lo necesita en los que se proporciona una dosificación que puede reducir o evitar una subexposición, p. ej., en sujetos de peso corporal bajo, y/o proporcionar otros beneficios.
CL2022001079A 2019-10-28 2022-04-28 Métodos y composiciones para el tratamiento del síndrome de rett CL2022001079A1 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201962927008P 2019-10-28 2019-10-28
US202063031201P 2020-05-28 2020-05-28

Publications (1)

Publication Number Publication Date
CL2022001079A1 true CL2022001079A1 (es) 2023-04-21

Family

ID=75715568

Family Applications (1)

Application Number Title Priority Date Filing Date
CL2022001079A CL2022001079A1 (es) 2019-10-28 2022-04-28 Métodos y composiciones para el tratamiento del síndrome de rett

Country Status (14)

Country Link
US (1) US20220339138A1 (es)
EP (1) EP4051308A4 (es)
JP (2) JP2022553888A (es)
KR (1) KR20220106982A (es)
CN (1) CN115335071A (es)
AU (1) AU2020376801A1 (es)
BR (1) BR112022008095A2 (es)
CA (1) CA3156680A1 (es)
CL (1) CL2022001079A1 (es)
CO (1) CO2022007501A2 (es)
IL (1) IL292617B1 (es)
MX (1) MX2022004785A (es)
TW (1) TW202116300A (es)
WO (1) WO2021086892A1 (es)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2023287750A1 (en) 2021-07-12 2023-01-19 Acadia Pharmaceuticals Inc. Crystalline forms of trofinetide
EP4539871A4 (en) * 2022-06-15 2025-09-24 Univ Ramot METHOD AND THERAPEUTIC AGENT FOR THE TREATMENT OF A DISEASE OR DISORDER ASSOCIATED WITH AN ALTERATION OF MITOCHONDRIAL CALCIUM FIRING RATE AND/OR HOMEOSTASIS

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
SI2667715T1 (sl) * 2011-01-27 2017-11-30 Neuren Pharmaceuticals Limited Zdravljenje motenj avtističnega spektra z uporabo glicil-l-2-metilprolil-l-glutaminske kisline
JP2016509030A (ja) * 2013-02-20 2016-03-24 セラバスク インコーポレーテッド ニトライトの医薬製剤及びそれらの使用
LT3024463T (lt) * 2013-07-25 2020-07-10 Neuren Pharmaceuticals Limited Neuroapsauginiai bicikliniai junginiai ir jų panaudojimo būdai autizmo spektro sutrikimų ir neuroliginių vystymosi sutrikimų gydymui
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

Also Published As

Publication number Publication date
CN115335071A (zh) 2022-11-11
BR112022008095A2 (pt) 2022-07-12
IL292617A (en) 2022-07-01
JP2022553888A (ja) 2022-12-26
KR20220106982A (ko) 2022-08-01
WO2021086892A1 (en) 2021-05-06
JP2025148461A (ja) 2025-10-07
EP4051308A1 (en) 2022-09-07
IL292617B1 (en) 2025-11-01
CO2022007501A2 (es) 2022-08-30
MX2022004785A (es) 2022-05-16
US20220339138A1 (en) 2022-10-27
AU2020376801A1 (en) 2022-06-09
EP4051308A4 (en) 2023-08-23
TW202116300A (zh) 2021-05-01
CA3156680A1 (en) 2021-05-06

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