ISCRIVITI ALLA REGISTRAZIONE PAZIENTI
Aiutaci a riunire pazienti, famiglie e ricercatori per ottenere a
migliore comprensione della sindrome di Malan.
What is a natural history study?
A natural history study is a type of research study that collects detailed information from patients to characterize the signs and symptoms of a disease and how the disease evolves over time.
Why is it important?
The data can help to:
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create a strong patient and research community
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allow healthcare/researchers to learn more about the syndrome
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develop standards of care
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inform drug development
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enable better data to use in clinical trials
Se tu o un membro della famiglia avete stata diagnosticata in qualsiasi parte del mondo con la sindrome di Malan, iscrivetevi al registro dei pazienti per dare un contributo importante alla ricerca sulla sindrome di Malan. Il database del registro consentirà una raccolta e un'analisi sistematiche di informazioni da individui con sindrome di Malan per aiutare a far progredire la comprensione della condizione.
Stiamo collaborando conSanford CoRDSper creare il registro. Sanford CoRDS supporta e consente alle comunità di malattie rare di creare solidi registri, fornendo ai researchers le informazioni di cui hanno bisogno per portare avanti la ricerca. Fare clic sulla casella CoRDS in basso per iscriversi.
How can I participate?
RARE-X
The Malan Syndrome Foundation is partnering with RARE-X, a 501(c)(3) nonprofit, to build a Data Collection Program for Malan families. When you participate in the Malan Syndrome Data Collection Program, you’ll help accelerate research and the development of new drugs, devices, and other therapies.
The data collection program powered by RARE-X involves completing validated, standardized surveys that ask questions on ALL systems of the body, from "head to toe". Both international and US-based families can enroll; surveys will be available in multiple languages starting early 2023.
Ciitizen
The Malan Syndrome Rare Patient Network powered by Ciitizen is a secure digital platform that organizes, summarizes and stores patient medical record information obtained from multiple hospitals and providers.
The information collected from the health records can help to inform a better understanding of Malan syndrome and further research toward potential treatments. All participants have access to their health records in Ciitizen and control who sees the information. Only US-based families can enroll at this time.
CoRDS
The Malan Syndrome Foundation partnered with Sanford CoRDS in 2019 to create a Malan syndrome registry. Participants will complete a questionnaire that has been developed specifically for Malan syndrome (disease-specific questionnaire). Both international and US-based families can enroll; surveys are available in English.
If you experience any issues with registering, please email info@malansyndrome.org.
