The SHARE because you CARE
ADNP Patient Registry Project
Participate in Research - Patient Powered Registry!
The SHARE because you CARE - ADNP Patient Powered Registry Project is an multi-level repository of patient reported data and on individuals diagnosed with ADNP Syndrome and their families/caregivers.
What is a patient-powered registry?
A patient registry is an organized system that uses observational study methods to collect uniform data, clinical and other, to evaluate specified outcomes for a population defined by a particular disease, condition, or exposure, and that serves as a predetermined scientific, clinical, or policy purpose. Our registry will be patient driven.
ADNP syndrome is a rare disease. A particular concern and road block for rare diseases 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.
What is the purpose?
Our registry will be patient driven with the purpose to engage our ADNP community in the data sharing process accelerating disease understanding, research and drug development. Our organization is working in collaboration with researchers around the world - so your data matters and will make a difference in understanding ADNP syndrome.
What are the benefits"
How will it work?
The registry will take place in multiple layers. To begin, the patient contact and general symptom log will be sent out. You will have access to fill out simple surveys and will have the opportunity to consent to be contacted by ADNP researchers. We will share only de-identified reports with you on a regular basis. If you are interested in participating please fill out the form beflow and refer to our listed privacy policy. (By filling out the form below you are not consenting to participate, you are expressing interest to be contacted when the registry
, an IRB approved registry questionnaire for natural history study data collection and supplemental questionnaires regarding clinical trial and clinical studies as needed. Over time, additional natural history study questionnaires will also be collected to identify the progression of the disease.
The ADNP Kids Research Foundation is dedicated to supporting research for education and treatment of ADNP syndrome and to improve the lives of those living with the disease. By creating a robust database and collaborating with world-leading scientific research experts, the ADNP Kids Research Foundation seeks to support an era of precision medicine that will dramatically improve the discovery of therapeutic treatments for ADNP Syndrome and make a meaningful difference in the lives of people with this severe and debilitating neurological genetic disorder.
By building the world’s most robust database of its kind, research teams from around the world will be able to expand their knowledge base and generate fresh insights and research initiatives supporting the development of more targeted and effective approaches in the treatment of ADNP Syndrome, including the FDA clinical trial for CP201/NAP. This data will ultimately assist the ADNP community in generating better patient care and quality of life for children and adults suffering with ADNP Syndrome.
Through our website we seek to educate patients and family members about the scientific findings from the registry and about advances in the diagnosis, treatment and management of ADNP Syndrome. This will allow participants to see ongoing published summaries of our findings as we build our database. It will produce valuable information for those affected and those treating current patients, it will assist future clinical and drug trials and contribute to the creation of a Natural History Study for ADNP.
WHY SHOULD YOU SHARE?
The SHARE because you CARE - ADNP Patient Registry Project is a patient driven multi-level registry. Your participation gives you the opportunity to help drive research by sharing information that will increase the understanding of the disorder, support and find treatments for ADNP syndrome. Your information is important and CAN made a difference!
As a grassroots non-profit organization with diagnosed ADNP children 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.
YOU CAN TAKE PART IN HELPING US ADVANCE THE UNDERSTANDING OF ADNP SYNDROME.
What is a patient-powered registry?
A patient registry is an organized system that uses observational study methods to collect uniform data, clinical and other, to evaluate specified outcomes for a population defined by a particular disease, condition, or exposure, and that serves as a predetermined scientific, clinical, or policy purpose. Our registry will be patient driven.
ADNP syndrome is a rare disease. A particular concern and road block for rare diseases 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.
What is the purpose?
Our registry will be patient driven with the purpose to engage our ADNP community in the data sharing process accelerating disease understanding, research and drug development. Our organization is working in collaboration with researchers around the world - so your data matters and will make a difference in 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 information regarding ongoing IRB approved research and clinical trials.
- One of the main concerns expressed in rare disease communities is the lack of shared patient-friendly data. We believe sharing de-identified data back with our patient community will engage and empower the ADNP community and move research forward.
- We aim to unify the patient and research community with this collaborative registry following strict data sharing guidelines.
How will it work?
The registry will take place in multiple layers. To begin, the patient contact and general symptom log will be sent out. You will have access to fill out simple surveys and will have the opportunity to consent to be contacted by ADNP researchers. We will share only de-identified reports with you on a regular basis. If you are interested in participating please fill out the form beflow and refer to our listed privacy policy. (By filling out the form below you are not consenting to participate, you are expressing interest to be contacted when the registry
, an IRB approved registry questionnaire for natural history study data collection and supplemental questionnaires regarding clinical trial and clinical studies as needed. Over time, additional natural history study questionnaires will also be collected to identify the progression of the disease.
The ADNP Kids Research Foundation is dedicated to supporting research for education and treatment of ADNP syndrome and to improve the lives of those living with the disease. By creating a robust database and collaborating with world-leading scientific research experts, the ADNP Kids Research Foundation seeks to support an era of precision medicine that will dramatically improve the discovery of therapeutic treatments for ADNP Syndrome and make a meaningful difference in the lives of people with this severe and debilitating neurological genetic disorder.
By building the world’s most robust database of its kind, research teams from around the world will be able to expand their knowledge base and generate fresh insights and research initiatives supporting the development of more targeted and effective approaches in the treatment of ADNP Syndrome, including the FDA clinical trial for CP201/NAP. This data will ultimately assist the ADNP community in generating better patient care and quality of life for children and adults suffering with ADNP Syndrome.
Through our website we seek to educate patients and family members about the scientific findings from the registry and about advances in the diagnosis, treatment and management of ADNP Syndrome. This will allow participants to see ongoing published summaries of our findings as we build our database. It will produce valuable information for those affected and those treating current patients, it will assist future clinical and drug trials and contribute to the creation of a Natural History Study for ADNP.
WHY SHOULD YOU SHARE?
The SHARE because you CARE - ADNP Patient Registry Project is a patient driven multi-level registry. Your participation gives you the opportunity to help drive research by sharing information that will increase the understanding of the disorder, support and find treatments for ADNP syndrome. Your information is important and CAN made a difference!
As a grassroots non-profit organization with diagnosed ADNP children 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.
YOU CAN TAKE PART IN HELPING US ADVANCE THE UNDERSTANDING OF ADNP SYNDROME.