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
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CURRENT RESEARCH GOING INTO 2023:
This year we began to focus on a wide range of project, including the launch of the first ever international ADNP Patient Registry, building the community by way of Parent Ambassadors, and most exciting - the advancement of our drug trial and numerous therapeutic options.
In the coming month, we will be sending out information on each one of our research projects in more detail.  They are all so exciting, we simply can't put it all down in one message!
RESEARCH:
Our FDA phase 2a drug trial held at Mount Sinai in New York, was completed and finally published.  Everyone is thrilled with the safety and efficacy shown in the first ever drug trail in actual patients with ADNP syndrome!   Our team has been working very hard with the FDA for the next phase, and just this month we received FDA IND approval to move forward!  
  • The 2023 portion of this project is $500,000.
Our project team at UC Davis MIND Institute in California have been working very hard at evaluating not 1, but 3 lead therapeutic gene therapy candidates for ADNP Syndrome.  These include testing an AAV vector, constructing an ASO, and working to test in parallel mice and iPSC derived neurons and brain organoids.
  • The 2023 portion of this project is $1.4 million dollars.
Our project team at RareBase in California also has given us some recent exciting news.  This drug repurposing project is focusing mainly on small molecules.  We screened thousands of drugs in brain cells in a disk and found more than 12 potential drug candidates and we are now moving into the preclinical stage of research. 
The 2023 portion of the preclinical research is $300,000
As you can see, we need to raise $2 million dollars for the 2023 year, with approximately another $2.5 million for IND enabling studies needed the following year for our gene therapy projects as well as our drug trail phase 3.  As a grassroots small charity, with a very low number of children diagnosed worldwide, at approximately 400, we are funding this research ourselves to accelerate the discovery of treatments and cures as fast as possible for not only our children living with ADNP, but we also believe that treatments we find will help other related neurological, developmental and autism disorders.  
Now more than ever, because of this great research and your support, there is HOPE!
With gratitude,


Sandra Bedrosian-Sermone
Founder, President, CEO
ADNP Kids Research Foundation
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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