Thalassemia
What is Thalassemia?
Thalassemia is a rare inherited disorder characterized by a reduced rate of synthesis of one of the globin chains that make up hemoglobin.
| Name | Description |
|---|---|
| Anemia | Anemia is a deficiency of hemoglobin. |
Last updated 28 Nov 2018, 04:47 AM
Last updated 4 Jul 2018, 08:48 PM
Last updated 5 Oct 2010, 02:31 AM
5 Oct 2010, 02:31 AM
Hi! I would love to hear from anyone who has been diagnosed with hasharon blood disorder.I'm told by RareShare that thalassemia is the most similiar disorder to hasharon.
| Title | Date | Link |
|---|---|---|
| FDA Approves REBLOZYL® (luspatercept-aamt) for the Treatment of Anemia in Adults With Beta Thalassemia Who Require Regular Red Blood Cell Transfusions | 11/17/2019 | |
| Newborn Screening Practices and Alpha-Thalassemia Detection — United States, 2016 | 09/12/2020 | |
| I've had a blood transfusion every 3 weeks since I was 2 months old due to a rare disease that can kill people in their 30s | 03/20/2022 | |
| Editas Medicine Gets FDA Rare Pediatric Disease Designation for EDIT-301 | 05/01/2022 | |
| bluebird bio Announces FDA Approval of ZYNTEGLO®, the First Gene Therapy for People with Beta-Thalassemia Who Require Regular Red Blood Cell Transfusions | 08/21/2022 | |
| The Most Expensive Drug in the US is a Rare-Disease Game Changer | 08/28/2022 | |
| FDA widens approval of Vertex’s CRISPR medicine to treat beta thalassemia | 01/17/2024 |
Clinical Trials
CoRDS, or the Coordination of Rare Diseases at Sanford, is based at Sanford Research in Sioux Falls, South Dakota. It provides researchers with a centralized, international patient registry for all rare diseases. This program allows patients and researchers to connect as easily as possible to help advance treatments and cures for rare diseases. The CoRDS team works with patient advocacy groups, individuals and researchers to help in the advancement of research in over 7,000 rare diseases. The registry is free for patients to enroll and researchers to access.
Enrolling is easy.
- Complete the screening form.
- Review the informed consent.
- Answer the permission and data sharing questions.
After these steps, the enrollment process is complete. All other questions are voluntary. However, these questions are important to patients and their families to create awareness as well as to researchers to study rare diseases. This is why we ask our participants to update their information annually or anytime changes to their information occur.
Researchers can contact CoRDS to determine if the registry contains participants with the rare disease they are researching. If the researcher determines there is a sufficient number of participants or data on the rare disease of interest within the registry, the researcher can apply for access. Upon approval from the CoRDS Scientific Advisory Board, CoRDS staff will reach out to participants on behalf of the researcher. It is then up to the participant to determine if they would like to join the study.
Visit sanfordresearch.org/CoRDS to enroll.
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Diagnosed by the Mayo Clinic in Rochester , MN , with SCLS in August 2011. This was my second trip to Mayo. The first trip was in August 2010 and resulted in a 2 week stay and a suspected / working...
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Article on Arrivant as potential therapy for sickle cell and thalassemia in RareShare news link section.
Created by RareshareTeam | Last updated 28 Nov 2018, 04:47 AM
See RareShare's news link section for latest rare disease news, including an article on use of gene therapy to treat thalassemia.
Created by RareshareTeam | Last updated 4 Jul 2018, 08:48 PM
hasharon blood disorder
Created by susanfv | Last updated 5 Oct 2010, 02:31 AM
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