Research helps us better understand diseases and can lead to advances in diagnosis and treatment. This section provides resources to help you learn about medical research and ways to get involved.
Support and advocacy groups can help you connect with other patients and families, and they can provide valuable services. Many develop patient-centered information and are the driving force behind research for better treatments and possible cures. They can direct you to research, resources, and services. Many organizations also have experts who serve as medical advisors or provide lists of doctors/clinics. Visit the group’s website or contact them to learn about the services they offer. Inclusion on this list is not an endorsement by GARD.
Living with a genetic or rare disease can impact the daily lives of patients and families. These resources can help families navigate various aspects of living with a rare disease.
These resources provide more information about this condition or associated symptoms. The in-depth resources contain medical and scientific language that may be hard to understand. You may want to review these resources with a medical professional.
NIH Hosts Rare Disease Day Event, Twitter Chat
January 24, 2019
Accelerating Cures in the Genomic Age: The Sickle Cell Example
December 12, 2018
Gene Editing Corrects Sickle Cell Mutation
October 26, 2016
Questions sent to GARD may be posted here if the information could be helpful to others. We remove all identifying information when posting a question to protect your privacy. If you do not want your question posted, please let us know. Submit a new question
What is the likelihood that a diagnosis of osteochondritis dissecans of the knee may have been caused by sickle cell disease and what are recommended treatment? See answer
What is the best pain management regimen for a 50-year-old with sickle beta thalassemia experiencing sickle cell crisis? Lately, the onset has become more frequent, about every three months. Is this normal? See answer