Sickle cell-beta-thalassemia disease syndrome

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Synonyms: HbS-beta-thalassemia syndrome

A rare genetic hemoglobinopathy that affects red blood cells both in the production of abnormal hemoglobin as well as the decreased synthesis of beta globin chains. Clinical manifestations depend on the amount of residual beta globin chains production and are similar to sickle cell disease including anemia vascular occlusion and its complications acute episodes of pain acute chest syndrome pulmonary hypertension sepsis ischemic brain injury splenic sequestration crisis and splenomegaly.

Data from Orphanet are used to provide information on a disease's name, synonym(s), and overview. Reference: Access aggregated data from Orphanet at Orphadata. Orphadata: Free access data from Orphanet. © INSERM 1999. Available on http://www.orphadata.org. Data version July 2024

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Advocacy Organizations

FELIS HEALTHCARE FOUNDATION INC

The mission is to provide free health care services, education, and training to the most vulnerable, poorest, and underserved.The vision is ever-increasing access to medical treatment, better education, and nutrition, especially for those unable to pay for the necessary products and services.This approach helps us transform the lives of millions of people and address data gaps on different issues,

HECI Medicine Incorporation

The objectives of HECI Medicine is to provide dependable access to affordable medicine and diagnostic tools to patients in Africa affected by rare diseases, collaborative with the scientific community to accelerate drug development for rare diseases therapies that are specific to populations in Africa, and collaborate with other rare disease organizations to ensure equitable access to health.

Hemostasis and Thrombosis Center of Nevada

The Hemostasis & Thrombosis Center of Nevada (HTCNV) is Nevada's only federally designated hemophilia treatment center (HTC). We provide care for benign inherited blood disorders specifically bleeding disorders, thrombotic disorders and sickle cell disease. Our mission is to provide access to compassion evidence based quality care to all patients in Nevada regardless of their ability to pay.

Hope Life Support & Empowerment Initiative

We are committed to building a society that is fully aware of their health status and to improve the health of people in communities where less disadvantaged children, youths, women and men lived

Journey to ExSCellence

The mission of Journey to ExSCellence is to is to invest in direct services and programming that improve the quality of life for Sickle Cell Warriors by bringing the gap between awareness and advocacy to direct support for people living with Sickle Cell Disease.

OPFORD Foundation

OPFORD.org (Open Platform for Rare Diseases) was conceived as a digital and online resource centre for orphan diseases with a primary focus of serving patients and caregivers by connecting them to curated resources regarding symptoms, causes, diagnosis and treatment. We have expanded our services to health counselling and guidance for patients and caregivers to prevent possible complications.

SICKLE CELL AWARENESS FOUNDATION CORP INT

To expand the genetic condition of sickle cell disease. To advocate for improved treatment services and accessibility to these services. To inform what parents, family members and the community can do to impact this disease condition. We seek to inspire progress for a much-needed cure for this disease.

Supporters of Families with Sickle Cell Disease, Inc

Our mission is to increase self-efficacy and improve the overall quality of life for individuals living with sickle cell and thalassemia, children, adults and their families within Oklahoma through systemic changes in patient care, disability policies, education, family support, economic self-sufficiency, awareness and advocacy.

TANZANIA SICKLECELL WARRIORS ORGANIZATION (TASIWA)

TANZANIA SICKLECELL WARRIORS ORGANISATION abbreviated as TASIWA, is Tanzanian non governmental and non profit organization which aims at improving the survival rates of sickle cell-born children by promoting better handling and management of sickle cell disease through various programs including education and advocacy which mostly done at the community level.

Uriel E. Owens Sickle Cell Disease Association of the Midwest

The mission of the Uriel E. Owens Sickle Cell Disease Association of the Midwest is to provide information and awareness about Sickle Cell Disease, to advocate for the needs of persons with Sickle Cell, and support research to find a cure. Besides education and awareness, the Association provides emergency financial assistance, referral services, state and national advocacy, and educational scholarships for sickle cell patients.

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Clinical Trials

For a list of clinical trials in this disease area, please click here.