BPAN Warriors Partners with Ciitizen to launch a groundbreaking Natural History Study to accelerate potential treatments for rare disease BPAN

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The parent-led research foundation, BPAN Warriors, announced today the launch of their groundbreaking BPAN Digital Natural History Study (NHS) in partnership with Ciitizen. The new BPAN NHS aims to break down the barriers to patient participation and accessibility in the current natural history study model, while accelerating rare disease drug development.

In a disease as complex as BPAN, accessing whole-health records allows us to see the entire story. Ciitizen provides us with the ability to rapidly assimilate and analyze 1000’s of points of data, across a large cohort of BPAN patients

The parent-led research foundation, BPAN Warriors, announced today the launch of their groundbreaking BPAN Digital Natural History Study (NHS) in partnership with Ciitizen, a Palo-Alto based healthcare technology company that helps patients get full control of their medical records so they can find better treatment options, participate in research and find clinical trials. The new BPAN NHS aims to break down the barriers to patient participation and accessibility in the current natural history study model, while accelerating rare disease drug development.

BPAN is a neurodegenerative disorder that leads to developmental delays, seizures, movement disorders and gradual loss of intellectual functioning. Clinical data has been and continues to be a source of incredible opportunity especially in the push toward precision medicine in rare disease. Understanding the natural history of a disease is an essential element to inform drug development. For rare diseases and patient advocacy organizations, prohibitive costs associated with existing NHS models and lack of participant access to study data, often limit patient engagement and provide insufficient natural history data, making it difficult to advance the care of children and adults living with a rare disease.

“A single lab report or a single test result only tells part of a story. In a disease as complex as BPAN, accessing whole-health records allows us to see the entire story. Ciitizen provides us with the ability to rapidly assimilate and analyze 1000’s of points of data, across a large cohort of BPAN patients,” said Sarah Chisholm, Founder of BPAN Warriors. “What we know today, may fundamentally change once we see our disease collectively and may provide us critical insights that will be key in advancing BPAN treatment therapies and improving patient care.”

For complex diseases such as BPAN, medical records may often be housed at multiple providers, hospitals, specialty clinics and labs. Through the Ciitizen BPAN NHS, medical records are collected, on the patient’s behalf from all medical providers designated by the patient during the short onboarding process, thereby significantly reducing the caregiver burden and providing access to a larger number of medical records.

Once Ciitizen has collected the patient health records, the Ciitizen BPAN NHS platform provides patients (caregivers) direct access to their consolidated, digitized data that summarizes their whole health experience and can be used to get second opinions or share with multiple providers to facilitate and improve overall patient care.
With consent, the anonymized data is then extracted and available for clinicians, researchers and biopharma to aid in research and therapy development. Researchers will be able to access large amounts of data to help determine clinical endpoints for potential treatments.

While aggregating patients’ health information is a critical first step, data standardization, accessibility and ultimately utility to the patient and research community are paramount. Understanding the patient's whole-health, shared symptoms, potential efficacy of pharmacological interventions in the treatment of symptoms, disease trajectory, and possible biomarkers, are all key in the drug development process.

BPAN Warriors joins forces with several rare disease groups including, SLC13A5 deficiency (TESS Research Foundation), SYNGAP1-related disorder (SynGAP Research Fund), Rett Syndrome (Rett Syndrome Research Trust), and FOXG1 syndrome (FOXG1 Research Foundation), who was recently awarded a $500k grant from the Chan Zuckerberg Initiative (CZI) to revolutionize the ability for patient-led advocacy groups to use machine learning to help accelerate rare disease drug development on the Ciitizen platform. Additional CNS diseases are slated for onboarding in 2021.

“Ciitizen was born when my sister Tania died from metastatic breast cancer, and I knew that she might have lived,” said Anil Sethi, Chief Executive Officer of Ciitizen. “One of the biggest issues in curing any disease, especially a rare disease, is access to better data - for better endpoint selection, to predict symptom variability, and to establish natural history of disease. We are inspired by the work of advocacy groups like BPAN Warriors and are honored to partner in their fight to find treatments”.

These advocacy groups understand the importance of both collaborative data practices and innovative technology that will redefine the drug development process for rare diseases. Ciitizen’s digital platform will augment the existing natural history dataset in disease communities where an NHS may already exist and provide a valuable and unique service to families and researchers. Collectively, applying machine learning to aggregated data across multiple rare diseases, with shared symptoms may yield evidence for multi-disease therapies.

About BPAN Warriors: Founded in 2018, BPAN Warriors is a 501(c)(3) parent-led global organization dedicated to funding collaborative science that will expedite a cure and therapies for children and adults who are afflicted with the rare, life-limiting, neurodegenerative, genetic disorder called BPAN (beta-propeller protein-associated neurodegeneration), caused by mutations of the WDR45 gene. BPAN is bi-phasic disease characterized by severe developmental, cognitive, and physical disabilities, epilepsy, autistic features, vision and hearing loss, complex movement disorders, and rapid cognitive decline in the second or third decade of life, marked by parkinsonism, dystonia and dementia. For more information, please visit http://www.bpanwarriors.org.

About Ciitizen: Ciitizen is a leading consumer health technology company whose mission is to help patients get full control of their medical records so they can find better treatment options, participate in research and find clinical trials. As a patient-directed care platform, Ciitizen believes patients should have full control of directing the use of their data, which serves as one of the richest sources of real world evidence, unlocking new options for patients and the community. For more information, contact research@ciitizen.com and visit http://ciitizen.com/research.

Media Contact:
Sarah Chisholm, Founder, Executive Director, BPAN Warriors
sarah@bpanwarriors.org
Virginie McNamar, Project Manager, Neurological and Rare Diseases, Ciitizen
virginie@ciitizen.com

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Sarah Chisholm
BPAN Warriors
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Virginie McNamar
Ciitizen
(317) 832-3072
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@bpanwarriors
since: 06/2017
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