Neurocrine Biosciences Leverages PicnicHealth to Reveal Critical Care Gaps for Patients with Rare Genetic Condition
Neurocrine Biosciences (NASDAQ:NBIX) and PicnicHealth have revealed key findings from their CAHtalog® Registry study focused on congenital adrenal hyperplasia (CAH). The research, conducted in collaboration with the CARES Foundation, identified that CAH patients require continuous, lifelong adjustments to glucocorticoid dosing to maintain condition control.
The study leverages PicnicHealth's AI-powered technology to curate medical records and enable remote participation, overcoming traditional challenges in rare disease research. The technology allows for data collection from multiple care locations while providing patients with an intuitive app featuring an AI assistant for medical record navigation.
Additional findings regarding treatment patterns and condition progression were presented at ENDO 2025, demonstrating the registry's value in advancing understanding of this rare genetic condition affecting adrenal gland function.
Neurocrine Biosciences (NASDAQ:NBIX) e PicnicHealth hanno presentato i risultati principali dello studio CAHtalog®, incentrato sull'iperplasia surrenalica congenita (CAH). La ricerca, condotta in collaborazione con la CARES Foundation, ha evidenziato che i pazienti con CAH necessitano di aggiustamenti continui e a vita del dosaggio di glucocorticoidi per mantenere il controllo della condizione.
Lo studio utilizza la tecnologia AI di PicnicHealth per raccogliere dati medici e permettere la partecipazione a distanza, superando le difficoltà tradizionali nella ricerca sulle malattie rare. Questa tecnologia consente la raccolta di dati da molteplici centri di cura offrendo ai pazienti un'app intuitiva con un assistente AI per la navigazione delle cartelle cliniche.
Ulteriori risultati riguardanti i modelli di trattamento e la progressione della patologia sono stati presentati a ENDO 2025, dimostrando il valore del registro nel migliorare la comprensione di questa rara condizione genetica che interessa la funzione delle ghiandole surrenali.
Neurocrine Biosciences (NASDAQ:NBIX) y PicnicHealth han revelado hallazgos clave del estudio CAHtalog®, centrado en la hiperplasia suprarrenal congénita (CAH). La investigación, realizada en colaboración con la Fundación CARES, identificó que los pacientes con CAH requieren ajustes continuos y de por vida en la dosificación de glucocorticoides para mantener el control de la condición.
El estudio aprovecha la tecnología impulsada por IA de PicnicHealth para recopilar registros médicos y permitir la participación remota, superando los desafíos tradicionales en la investigación de enfermedades raras. La tecnología permite la recopilación de datos desde múltiples centros de atención y ofrece a los pacientes una aplicación intuitiva con un asistente de IA para navegar sus registros médicos.
Se presentaron hallazgos adicionales sobre los patrones de tratamiento y la progresión de la condición en ENDO 2025, demostrando el valor del registro para avanzar en la comprensión de esta rara condición genética que afecta la función de las glándulas suprarrenales.
Neurocrine Biosciences (NASDAQ:NBIX)와 PicnicHealth가 선천성 부신과형성증(CAH)을 중심으로 한 CAHtalog® 등록 연구의 주요 결과를 발표했습니다. CARES 재단과 협력하여 진행된 이번 연구는 CAH 환자들이 상태를 관리하기 위해 평생 지속적으로 글루코코르티코이드 용량을 조절해야 한다는 점을 밝혔습니다.
이 연구는 PicnicHealth의 AI 기반 기술을 활용해 의료 기록을 수집하고 원격 참여를 가능하게 하여 희귀질환 연구의 전통적 어려움을 극복했습니다. 이 기술은 여러 치료 기관에서 데이터를 수집할 수 있으며, 환자들에게는 의료 기록 탐색을 돕는 AI 어시스턴트가 탑재된 직관적인 앱을 제공합니다.
치료 패턴과 질환 진행에 관한 추가 결과는 ENDO 2025에서 발표되어, 이 희귀 유전 질환인 부신 기능 장애에 대한 이해를 높이는 데 등록 연구의 가치를 입증했습니다.
Neurocrine Biosciences (NASDAQ:NBIX) et PicnicHealth ont dévoilé des résultats clés de leur étude CAHtalog® portant sur l'hyperplasie congénitale des surrénales (CAH). Cette recherche, réalisée en collaboration avec la CARES Foundation, a identifié que les patients atteints de CAH nécessitent des ajustements continus et à vie des doses de glucocorticoïdes pour maintenir le contrôle de leur maladie.
L'étude utilise la technologie IA de PicnicHealth pour compiler les dossiers médicaux et permettre une participation à distance, surmontant ainsi les défis traditionnels de la recherche sur les maladies rares. Cette technologie permet la collecte de données provenant de multiples centres de soins tout en offrant aux patients une application intuitive avec un assistant IA pour naviguer dans leurs dossiers médicaux.
Des résultats supplémentaires concernant les schémas de traitement et la progression de la maladie ont été présentés lors de ENDO 2025, démontrant la valeur du registre pour faire avancer la compréhension de cette rare maladie génétique affectant la fonction des glandes surrénales.
Neurocrine Biosciences (NASDAQ:NBIX) und PicnicHealth haben wichtige Ergebnisse ihrer CAHtalog®-Registerstudie zur kongenitalen adrenalen Hyperplasie (CAH) vorgestellt. Die in Zusammenarbeit mit der CARES Foundation durchgeführte Forschung zeigte, dass CAH-Patienten kontinuierliche, lebenslange Anpassungen der Glukokortikoid-Dosierung benötigen, um die Krankheitskontrolle aufrechtzuerhalten.
Die Studie nutzt die KI-gestützte Technologie von PicnicHealth, um medizinische Unterlagen zu kuratieren und eine Fernteilnahme zu ermöglichen, wodurch traditionelle Herausforderungen in der Forschung seltener Krankheiten überwunden werden. Die Technologie erlaubt die Datenerfassung an mehreren Behandlungsorten und bietet den Patienten eine intuitive App mit einem KI-Assistenten zur Navigation durch medizinische Unterlagen.
Weitere Erkenntnisse zu Behandlungsmustern und Krankheitsverlauf wurden auf der ENDO 2025 präsentiert und zeigen den Wert des Registers zur Vertiefung des Verständnisses dieser seltenen genetischen Erkrankung, die die Funktion der Nebennieren betrifft.
- Implementation of AI-powered technology enables more efficient rare disease research
- Remote participation capability increases patient accessibility and engagement
- Collaboration with CARES Foundation enhances patient recruitment and engagement
- Study reveals ongoing challenges in maintaining stable treatment for CAH patients
- Patients require continuous glucocorticoid dosing adjustments throughout life
Insights
Neurocrine's CAHtalog Registry reveals critical treatment insights for a rare genetic disorder, showcasing potential commercial opportunity in an underserved market.
Neurocrine Biosciences' collaboration with PicnicHealth has yielded valuable clinical insights into congenital adrenal hyperplasia (CAH), a rare genetic condition affecting adrenal gland function. The research identified that patients require lifelong, continuous adjustments to glucocorticoid dosing to maintain condition control, highlighting a significant unmet need in this patient population.
This finding is particularly meaningful from a clinical development perspective. By identifying the challenge of maintaining stable condition control with current treatment approaches, Neurocrine has established a clear target for potential therapeutic innovation. The company appears to be building a comprehensive understanding of CAH treatment patterns and disease progression, with additional findings presented at ENDO 2025.
The CAHtalog® Registry demonstrates Neurocrine's strategic approach to rare disease drug development by leveraging technology-enabled research. Using AI-powered analysis and remote participation models addresses the inherent challenges in studying geographically dispersed rare disease populations. This patient-centric approach to data collection, facilitated by their partnership with the CARES Foundation, enables more efficient research while maintaining patient engagement.
For Neurocrine, this initiative represents a methodical expansion of their neuroscience and endocrinology portfolio into a well-defined rare disease with documented treatment gaps. By establishing themselves as leaders in understanding CAH management, they're positioned to potentially develop more optimized therapeutic approaches for this underserved patient population.
Technology-enabled research approach and AI-powered analysis identify unmet needs in patients with congenital adrenal hyperplasia
CAHtalog® was founded in 2021 by PicnicHealth and Neurocrine, in collaboration with the CARES Foundation, to establish a
"This study shows how researchers can minimize patient burden while still gathering valuable, long-term insights to answer their research questions," says Dan Drozd, MD, MSc, chief medical officer at PicnicHealth. "New technologies, including AI, make it possible to integrate data from across patients' medical records, expanding knowledge on this condition and providing direct benefits to participating patients."
PicnicHealth conducts non-interventional research by collecting medical records from anywhere patients receive care and enabling patients to complete research activities through an intuitive app. The app also features an AI assistant that empowers patients to navigate their medical records with ease. This novel approach to registries helps life sciences companies answer their research questions more efficiently while fostering long-term patient engagement. The CARES Foundation proved crucial for successfully recruiting and engaging CAH patients in the study.
"We're always looking for new ways to support our patients," says Dina Matos, executive director of the CARES Foundation. "This registry gives our community the opportunity to contribute their valuable data to advance CAH research, while also empowering them with tools to better manage their condition throughout their health journey."
In addition to this key finding on glucocorticoid dosing, additional results from the CAHtalog® registry on treatment patterns and condition progression were presented earlier this week at ENDO 2025 in Treatment Patterns and Changes in Health States in Patients with Classic Congenital Adrenal Hyperplasia: An Analysis of Data from the CAHtalog® Registry and in the poster ADRENAL (EXCLUDING MINERALOCORTICOIDS): Adrenal Insufficiency and CAH III.
Patients interested in joining the CAHtalog® registry and advancing critical insights like these can learn more here.
CAHtalog® is a registered trademark of Neurocrine Biosciences, Inc.
About PicnicHealth
PicnicHealth is a leading health technology company dedicated to advancing the next generation of non-interventional research. To date, the company's direct-to-patient approach and innovative AI and technology platform have enabled 12 of the top 20 largest life science companies to run more efficient non-interventional research. PicnicHealth has given tens of thousands of patients access to tools and virtual care services to simplify their care journey. PicnicHealth was recently named one of the World's Best Digital Health Companies by Newsweek, "Best MedTech Startup" by MedTech Breakthrough, and "Best AI-enabled Life Sciences Solution" by Global Health & Pharma. The future is here with PicnicHealth. To learn more, visit picnichealth.com.
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SOURCE PicnicHealth
FAQ
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