FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases

Launched by XCURES · Aug 1, 2024

Keywords

ClinConnect Summary

The FLOWER trial is a nationwide study that aims to gather important information about rare diseases, such as Alpha-Thalassemia, Beta-Thalassemia, and many others. This study is completely virtual, meaning you can participate from the comfort of your own home. If you have a rare disease—defined as affecting fewer than 200,000 people in the U.S.—and are willing to give your consent, you might be eligible to join. Participants will need to allow the study team to access their medical records, which will be collected through secure electronic methods to help researchers understand these conditions better.

If you decide to participate, you can expect to provide some basic information and allow your medical records to be accessed. This will help the researchers compile a comprehensive dataset that can be used to improve care and treatment options for those with rare diseases. The study is currently recruiting participants of all ages and genders, and your involvement will contribute valuable knowledge to the medical community.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• * Any person with a known or suspected rare disease, defined by their prevalence of fewer than 200,000 individuals nationwide. Diseases include but are not limited to:
• • Alpha- or Beta- Thalassemia Amyloidosis Amyotrophic Lateral Sclerosis (ALS) Creutzfeldt-Jakob disease (CJD) Cystic Fibrosis (CF) Duchenne Muscular Dystrophy (DMD) Early-onset Alzheimer's Disease Ehlers-Danlos Syndrome (EDS) Huntington's Disease (HD) Gaucher Disease GM1 Gangliosidosis Myasthenia Gravis Pompe Disease Sickle Cell Disease Transthyretin Amyloid Cardiomyopathy (ATTR-CM) Transthyretin Amyloid Polyneuropathy (ATTR-PN)
• • - Patients or their legally-authorized representative must be willing and able to provide informed consent (and assent, if applicable). Deceased persons may participate via consent of their legally-authorized representative in accordance with applicable Federal and state laws
• Exclusion Criteria:
• • Patient or LAR is unable to provide informed consent.
• • Patient resides in a country other than the United States and is unable to provide access to medical records.