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registro de pacientes

Registro mundial de pacientes con síndrome de Leigh

Ayúdenos a reunir a pacientes, familias e investigadores para comprender mejor el síndrome de Leigh. Si usted o un miembro de su familia ha sido diagnosticado con el Síndrome de Leigh en cualquier parte del mundo, inscríbase en el registro de pacientes para hacer una contribución importante a la investigación del Síndrome de Leigh.  

 

La Fundación Cure Mito se está asociando con Sanford CoRDS para crear el registro. Sanford CoRDS apoya y permite a las comunidades de enfermedades raras crear registros sólidos, brindando a los investigadores la información que necesitan para impulsar la investigación.  

Para obtener más información sobre el registro, lea Aprobado por el IRB  Preguntas frecuentes .  

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To hear an audio recording please click below:

Cure Mito LS registry recording 02162022
00:00 / 04:59

Alignment and Interoperability of Leigh Syndrome Registry Data with Regulatory Submission Standards

Presented at PHUSE/FDA Computational Science Symposium, September 2022

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.

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Building a Worldwide Community - Leigh Syndrome Global Patient Registry 

Presented 2022 NORD Rare Diseases and Orphan Products Breakthrough Summit on October 17-18, 2022.

To download PDF of the poster in full size please click here.

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