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 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
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.
- The 2023 portion of this project is $1.4 million dollars.
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
