ADNP Kids Research Foundation
  • About
    • What is ADNP
    • Our Story
    • Our Mission
    • Our Team
    • Medical and Scientific Advisory Board
    • 501(c)(3) Determination
    • Inclusivity Statement
  • RESEARCH
    • Research Projects
    • Research Strategy
    • Ketamine Trial
    • Ketamine Trial Publication
    • Ketamine Update
    • Path to a cure
    • NET STUDY
    • publications
  • Families
    • Newly Diagnosed
    • ADNP Patient Registry
    • Contact Registry
    • Understanding the Basics
    • Worldwide Parent Ambassadors
    • contact us
    • ADNP Family Conference >
      • HIGHLIGHTS- ADNP Syndrome Family Conference
      • TSA Travel Help
      • #CureADNP
  • NEWS
    • NET Collaboration Study
    • Ketamine Trial Phase 2 Announcement
    • Mount Sinai Launches First Drug Trial for ADNP Syndrome
    • Seaver Midtown Partnership
    • Biomarker Discovered
    • NEWS - p.Tyr719* Case Study
    • NEWS HIGHLIGHTS- ADNP Syndrome Family Conference
  • ADNP STORE
  • DONATE/SUPPORT
    • Warrior Fun Run 2022 >
      • Past Fun Run Pictures
      • Sponsorship Opportunity
    • Boston Marathon
    • Third Party Fundraising
  • About Us new

FUNDING SCIENCE.  FINDING HOPE.

STRATEGIC RESEARCH ROADMAP
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​PATH TO A CURE
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1.  BUILD ADNP "MODELS"
  • Mouse Models (Joe Buxbaum, Seaver Center c.2188 knockin mouse held at Mount Sinai and Frank Kooy, Belgium mouse at JAX Labs)
  • Patient specific pluripotent stem cell models (5 ADNP patient iPSCs are held at Mount Sinai Seaver Center as well as a line at University of Washington)
  • Zebrafish Models
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2.  STUDY THE MODELS
  • Understand the impact of ADNP mutations on the brain and body:  Molecular, cellular and behavioral
  • Identify robust biomarkers and endpoints (diagnostic biomarker identified, visual evoke change biomarker, and 2 types of methylation in ADNP) (endpoints are being validated through phase 2 FDA ketamine drug trial)
  • Learn what time-points are effective for treatments 
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3.  TEST THERAPIES ON MODELS
  • Screen approved drugs
  • Identify and screen various therapies including: Gene Therapies, ASOs. etc.
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4.  SAFETY STUDIES
  • Conduct toxicology and efficacy of new successful drugs and therapies on wild animal types 
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5.  PATIENT CLINICAL TRIALS
  • ​FDA and International Regulatory Agency review
  • (*2021 - Conducting efficacy and safety phase 1/2 FDA low dose ketamine drug trial)

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Privacy Policy  |  Terms of Services  |  Other Policies  
All rights reserved. No portion of the content of this website may be reproduced in any manner without the written permission of the author/creator.   Any information used from this website must also have credit noted to ADNP Kids Research Foundation / www.adnpfoundation.org.
Disclosure: This page represents information known to the best of our knowledge.  Given the sensitivity and fast changing pace of research and drug development, some of the information posted on our website www.adnpfoundation.org may be inaccurate or not complete, therefor this information should be confirmed by the reader. Additionally, the medical information on this website is not substitute for personal diagnosis or medical care advise. Families and patients should consult a medically qualified clinician in all matters relating to genetic diagnosis, disease management and health. Information on ADNP research is a very fast-moving field and while the information here is believed to be the best available at the time of publication, some facts may later be updated or change. Please see additional disclosures on our PRIVACY POLICY page.
  • About
    • What is ADNP
    • Our Story
    • Our Mission
    • Our Team
    • Medical and Scientific Advisory Board
    • 501(c)(3) Determination
    • Inclusivity Statement
  • RESEARCH
    • Research Projects
    • Research Strategy
    • Ketamine Trial
    • Ketamine Trial Publication
    • Ketamine Update
    • Path to a cure
    • NET STUDY
    • publications
  • Families
    • Newly Diagnosed
    • ADNP Patient Registry
    • Contact Registry
    • Understanding the Basics
    • Worldwide Parent Ambassadors
    • contact us
    • ADNP Family Conference >
      • HIGHLIGHTS- ADNP Syndrome Family Conference
      • TSA Travel Help
      • #CureADNP
  • NEWS
    • NET Collaboration Study
    • Ketamine Trial Phase 2 Announcement
    • Mount Sinai Launches First Drug Trial for ADNP Syndrome
    • Seaver Midtown Partnership
    • Biomarker Discovered
    • NEWS - p.Tyr719* Case Study
    • NEWS HIGHLIGHTS- ADNP Syndrome Family Conference
  • ADNP STORE
  • DONATE/SUPPORT
    • Warrior Fun Run 2022 >
      • Past Fun Run Pictures
      • Sponsorship Opportunity
    • Boston Marathon
    • Third Party Fundraising
  • About Us new