реестр пациентов
Interoperability of Leigh Syndrome Patient Registry Data with Regulatory Submission Standards
Our paper: "Interoperability of Leigh Syndrome Patient Registry Data with Regulatory Submission Standards" is published in the Journal of the Society for Clinical Data Management: https://www.jscdm.org/article/id/244
Leigh Syndrome Global Patient Registry: Uniting Patients and Researchers Worldwide
Our paper: "Leigh Syndrome Global Patient Registry: Uniting Patients and Researchers Worldwide" is published in the Orphanet Journal of Rare Diseases!
To our knowledge this is the first paper about a global patient registry for Leigh syndrome, with many of the reported outcomes reported for the first time. It may also be a first truly international registry in mitochondrial disease with 70% of participants residing outside the United States.
Please read the full paper HERE
Leigh Syndrome Global Patient Registry: Uniting Patients and Researchers Worldwide
Our paper: "Leigh Syndrome Global Patient Registry: Uniting Patients and Researchers Worldwide" is published in the Orphanet Journal of Rare Diseases!
To our knowledge this is the first paper about a global patient registry for Leigh syndrome, with many of the reported outcomes reported for the first time. It may also be a first truly international registry in mitochondrial disease with 70% of participants residing outside the United States.
Please read the full paper HERE
Leigh Syndrome Global Patient Registry
Presented at Euromit, June 2023
To download PDF of the poster in full size please click here.
Building a Worldwide Community - Leigh Syndrome Global Patient Registry
Presented at NORD Rare Diseases and Orphan Products Breakthrough Summit on October 17-18, 2022.
Presented at Mitochondrial Medicine - Therapeutic Development conference in November 2022.
Click HERE to view short overview on YouTube.
To download PDF of the poster in full size please click here.
For a 2 minute video presentation please click here.
Alignment and Interoperability of Leigh Syndrome Registry Data with Regulatory Submission Standards
Presented at PHUSE/FDA Computational Science Symposium, September 2022.
Presented at Mitochondrial Medicine - Therapeutic Development conference in November 2022.
Click HERE to view short overview on YouTube.
Authors: Sophia Zilber, Pallavi Bakare, Kasey Woleben, Saima Kayani, Parag Shiralkar, and Japhanya Bhupathi
Abstract:
The patient data is collected at registries for getting a real-world view of patient reported outcomes, to better understand the disease, and facilitate clinical trials recruitment. Establishment of interoperability of registry data with acceptable submission standards like CDISC is essential in order to accelerate the development of therapies and is a critical milestone in case of rare diseases.
Cure Mito Foundation and Sumptuous Data Sciences, LLC conducted a pilot project for establishment of such interoperability of Leigh Syndrome patient-populated registry data with CDISC standards. This poster presentation provides an overview of the outcome of this pilot project. The poster provides overview of the current data collection practices of patient registry data used by Cure Mito Foundation, provides methodical steps executed to convert such data into CDISC requirements, and provides further assessments regarding conversion of patient-populated data to CDISC and its applicability to other rare disease patient registries.
To download PDF of the poster in full size please click here.
For a 2 minute video presentation please click here.
Глобальный реестр пациентов с синдромом Ли
Помогите нам объединить пациентов, семьи и исследователей, чтобы лучше понять синдром Ли. Если у вас или члена вашей семьи был диагностирован синдром Ли где-либо в мире, пожалуйста, зарегистрируйтесь в реестре пациентов, чтобы внести важный вклад в исследование синдрома Ли.
Фонд Cure Mito Foundation сотрудничает с Sanford CoRDS для создания реестра. Sanford CoRDS поддерживает и позволяет сообществам людей, страдающих редкими заболеваниями, создавать надежные реестры, предоставляя исследователям информацию, необходимую им для продвижения исследований.
Дополнительные сведения о реестре см. в документе, одобренном IRB. Часто задаваемые вопросы .
To hear an audio recording please click below:
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Фонд Cure Mito занимается развитием образования и исследований синдрома Ли и митохондриальных заболеваний. Наша миссия состоит в том, чтобы дать семьям возможность объединиться для достижения общей цели поиска лечения и, в конечном итоге, лекарства от этой разрушительной болезни. Пациенты находятся в центре всего, что мы делаем.
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