The SHARE because you CARE
ADNP Patient Research Study
Participate in our global Patient Powered Research Study!
Share Because You Care!
The SHARE because you CARE - ADNP Patient Research Project is the first-ever global patient registry of patient reported data in conjunction with the first ever natural history study for ADNP Syndrome patients! It is designed to collect medical information and patient-reported information about ADNP Syndrome.
By creating a robust database and collaborating with world-leading scientific research experts, including Mount Sinai Seaver Autism and Nuerodevelopmental Treatment Center New York, University of Washington Seattle, University of Antwerp Belgium, and Coronis Nuerosciences Israel, the ADNP Kids Research Foundation seeks to expand knowledge and generate insights and research initiatives supporting the accelerated development of targeted and effective approaches in the treatment of ADNP Syndrome, including the potential orphan drug clinical trial for CP201/NAP. This data will ultimately assist the ADNP community in generating a natural history study, patient outcome reports, better patient care and make a meaningful difference in the lives of people with this severe and debilitating neurological genetic disorder.
Why Should You SHARE?
Your participation gives you the opportunity to help drive research by sharing information that is important to you and will increase the understanding of the disorder. Your information is important and CAN made a difference now and for future generations to come!
Why Do We CARE?
As a grassroots non-profit organization linked to diagnosed ADNP children/patients located all over the world, we understand that it is very difficult to gather usable information. In addition, the rarity of the disorder makes it difficult to conduct large clinical trials. To address these deficiencies, the ADNP Kids Research Foundation invites you to join the The SHARE because you CARE - ADNP Patient Registry Project so that your data can be included in this valuable research.
What is a patient-powered registry?
A Patient Research Registry is an organized system that collects uniform patient/parent reported data, clinical and other, to evaluate specified outcomes for a population defined by a particular disease or condition.
What is our Registry purpose?
Our registry will be patient driven with the purpose to engage our ADNP community in accelerating disease understanding, research and drug development. ADNP syndrome is a rare disease without a uniform global Patient Registry. A particular concern and road block for good research is when multiple small site patient registries exist for a single rare condition, resulting in small data sets for each registry. These types of registries are less able to draw valid conclusions for the population. Our organization is working in collaboration with researchers around the world in an effort to share information, and work together, in a way that will make a meaningful difference for ADNP research. understanding ADNP syndrome.
What are the benefits:
In addition to these benefits, one of our main concerns is the lack of "shared" patient-friendly data. We believe sharing "de-identified" group data reports back with the patient community will engage and empower the ADNP community by giving needed information to the families who deal with ADNP on a daily basis. We hope to move unify the patient and research community with this registry following strict data sharing guidelines.
?
The registry will take place in multiple questionnaire surveys. As an example, there will be surveys regarding patient contact and general diagnosis information, general symptom surveys and also more detailed surveys pertaining to a specific area of concern such as behavioral, gastro-intestinal, sleep, education, etc. Surveys for natural history study data collection and supplemental questionnaires regarding clinical trial and clinical studies will also be added as needed. Over time, additional questionnaires will also be collected to identify the progression of the disease.
The SHARE because you CARE - ADNP Patient Research Project is the first-ever global patient registry of patient reported data in conjunction with the first ever natural history study for ADNP Syndrome patients! It is designed to collect medical information and patient-reported information about ADNP Syndrome.
By creating a robust database and collaborating with world-leading scientific research experts, including Mount Sinai Seaver Autism and Nuerodevelopmental Treatment Center New York, University of Washington Seattle, University of Antwerp Belgium, and Coronis Nuerosciences Israel, the ADNP Kids Research Foundation seeks to expand knowledge and generate insights and research initiatives supporting the accelerated development of targeted and effective approaches in the treatment of ADNP Syndrome, including the potential orphan drug clinical trial for CP201/NAP. This data will ultimately assist the ADNP community in generating a natural history study, patient outcome reports, better patient care and make a meaningful difference in the lives of people with this severe and debilitating neurological genetic disorder.
Why Should You SHARE?
Your participation gives you the opportunity to help drive research by sharing information that is important to you and will increase the understanding of the disorder. Your information is important and CAN made a difference now and for future generations to come!
Why Do We CARE?
As a grassroots non-profit organization linked to diagnosed ADNP children/patients located all over the world, we understand that it is very difficult to gather usable information. In addition, the rarity of the disorder makes it difficult to conduct large clinical trials. To address these deficiencies, the ADNP Kids Research Foundation invites you to join the The SHARE because you CARE - ADNP Patient Registry Project so that your data can be included in this valuable research.
What is a patient-powered registry?
A Patient Research Registry is an organized system that collects uniform patient/parent reported data, clinical and other, to evaluate specified outcomes for a population defined by a particular disease or condition.
What is our Registry purpose?
Our registry will be patient driven with the purpose to engage our ADNP community in accelerating disease understanding, research and drug development. ADNP syndrome is a rare disease without a uniform global Patient Registry. A particular concern and road block for good research is when multiple small site patient registries exist for a single rare condition, resulting in small data sets for each registry. These types of registries are less able to draw valid conclusions for the population. Our organization is working in collaboration with researchers around the world in an effort to share information, and work together, in a way that will make a meaningful difference for ADNP research. understanding ADNP syndrome.
What are the benefits:
- To characterize and describe our patient population as a whole.
- To facilitate the development of international best practices and care management guidelines and recommendations for improving quality of life.
- To provide needed information for ongoing and future IRB approved research and clinical trials.
- To generate patient needs and drug trial patient outcome studies.
In addition to these benefits, one of our main concerns is the lack of "shared" patient-friendly data. We believe sharing "de-identified" group data reports back with the patient community will engage and empower the ADNP community by giving needed information to the families who deal with ADNP on a daily basis. We hope to move unify the patient and research community with this registry following strict data sharing guidelines.
?
The registry will take place in multiple questionnaire surveys. As an example, there will be surveys regarding patient contact and general diagnosis information, general symptom surveys and also more detailed surveys pertaining to a specific area of concern such as behavioral, gastro-intestinal, sleep, education, etc. Surveys for natural history study data collection and supplemental questionnaires regarding clinical trial and clinical studies will also be added as needed. Over time, additional questionnaires will also be collected to identify the progression of the disease.
Take Part and SHARE because you CARE!
If you would like to take part in helping us advance the understanding of ADNP Syndrome, please review our Contact and General Information Informed Consent.
(CONTACT AND GENERAL INFORMATION INFORMED CONSENT)
If you would like to take part in helping us advance the understanding of ADNP Syndrome, please review our Contact and General Information Informed Consent.
(CONTACT AND GENERAL INFORMATION INFORMED CONSENT)