IL326528A - טיפול בחסר enpp1וחסר abcc6 בילדים - Google Patents

טיפול בחסר enpp1וחסר abcc6 בילדים

Info

Publication number
IL326528A
IL326528A IL326528A IL32652826A IL326528A IL 326528 A IL326528 A IL 326528A IL 326528 A IL326528 A IL 326528A IL 32652826 A IL32652826 A IL 32652826A IL 326528 A IL326528 A IL 326528A
Authority
IL
Israel
Prior art keywords
deficiency
abcc6
children
treatment
enpp1
Prior art date
Application number
IL326528A
Other languages
English (en)
Original Assignee
Inozyme Pharma Inc
Biomarin 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 Inozyme Pharma Inc, Biomarin Pharm Inc filed Critical Inozyme Pharma Inc
Publication of IL326528A publication Critical patent/IL326528A/he

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
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • 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
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/12—Drugs for disorders of the urinary system of the kidneys
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A61P19/04—Drugs for skeletal disorders for non-specific disorders of the connective tissue
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y306/00—Hydrolases acting on acid anhydrides (3.6)
    • C12Y306/01—Hydrolases acting on acid anhydrides (3.6) in phosphorus-containing anhydrides (3.6.1)
    • C12Y306/01009—Nucleotide diphosphatase (3.6.1.9), i.e. nucleotide-pyrophosphatase

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Epidemiology (AREA)
  • Genetics & Genomics (AREA)
  • Biochemistry (AREA)
  • Zoology (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Urology & Nephrology (AREA)
  • Biomedical Technology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Dermatology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
IL326528A 2023-08-22 2024-08-21 טיפול בחסר enpp1וחסר abcc6 בילדים IL326528A (he)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202363578137P 2023-08-22 2023-08-22
PCT/US2024/043242 WO2025042995A1 (en) 2023-08-22 2024-08-21 Treatment of enpp1 deficiency and abcc6 deficiency in children

Publications (1)

Publication Number Publication Date
IL326528A true IL326528A (he) 2026-04-01

Family

ID=94732792

Family Applications (1)

Application Number Title Priority Date Filing Date
IL326528A IL326528A (he) 2023-08-22 2024-08-21 טיפול בחסר enpp1וחסר abcc6 בילדים

Country Status (5)

Country Link
CN (1) CN121816188A (he)
AU (1) AU2024326788A1 (he)
CO (1) CO2026002943A2 (he)
IL (1) IL326528A (he)
WO (1) WO2025042995A1 (he)

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20250078598A (ko) * 2015-05-19 2025-06-02 예일 유니버시티 병리학적 석회화 병태를 치료하기 위한 조성물, 및 이의 사용 방법
AU2021383830A1 (en) * 2020-11-19 2023-06-22 Biomarin Pharmaceutical Inc. Treatment of enpp1 deficiency and abcc6 deficiency

Also Published As

Publication number Publication date
WO2025042995A9 (en) 2025-10-16
CN121816188A (zh) 2026-04-07
WO2025042995A1 (en) 2025-02-27
AU2024326788A1 (en) 2026-03-05
CO2026002943A2 (es) 2026-04-16

Similar Documents

Publication Publication Date Title
PL3799848T3 (pl) Wyroby chłonne i sposoby wytwarzania
AU304881S (en) Floor treatment device
IL326528A (he) טיפול בחסר enpp1וחסר abcc6 בילדים
AU2021383830A9 (en) Treatment of enpp1 deficiency and abcc6 deficiency
HUE067894T2 (hu) Mirdametinib és lifirafenib együttes beadása rákos megbetegedések kezelésében történõ alkalmazásra
EP4457239A4 (en) MODIFIED IMMUNE CELLS WITH ENHANCED POWER AND THEIR USES IN IMMUNOTHERAPY
CA3302510A1 (en) Treatment of enpp1 deficiency and abcc6 deficiency in children
IL288806A (he) מוטנטים מיוצבים של " quorum quenching lactonase" ושימושיהם בטיפול בפתוגנים
EP4247406A4 (en) TREATMENT OF ENPP1 DEFICIENCY AND ABCC6 DEFICIENCY
IL309249A (he) מצמידים הכוללים פוספואנטיגנים והשימוש בהם בטיפול
GB2621505B (en) Pyrrolopyridone derivatives useful in the treatment of inflammatory disorders, immune disorders and cancer
IL306026A (he) יכולת טרנספורמציה מוגברת והשראת האפלואיד בצמחים
ES3062513T3 (en) Factor ix variants and uses thereof in therapy
IL319234A (he) תרכובות פירזולילסולפונאמיד והשימוש שלהן בתרפיה
IL290430A (he) שיטה להגברת יעילות ויציבות של מרכיבים בתרחיפים
IL307497A (he) משטרי מינון וורוסיקליב ושיטות טיפול הכוללות אותם
EP4504924A4 (en) TREATMENT FOR ENPP1 DEFICIENCY AND ABCC6 DEFICIENCY
GB202306004D0 (en) Diaper changing tables and methods of using thereof
EP3955752A4 (en) AROMATIC ALKAMIDES AND METHODS OF USE THEREOF IN FLAVOR MODULATION
PT4284843T (pt) Sialósidos inovadores e sua utilização em terapia
CA3296320A1 (en) Nanoparticle (np)-enhanced expression of aquaporin-4 channels and water transport in human astrocytes
IL304640A (he) חלקיקי rnai ושיטות לשימוש בהם בחקלאות
GB202316080D0 (en) Improvements in or relating to devices and methods
IL315785A (he) פורמולציה משופרת של תיופורין ושיטות טיפול
ZA202210311B (en) Coronavirus therapeutics and treatment methods