Ads
related to: sickle cell disease treatment- Safety and Side Effects
Review the Safety Profile
And Potential Side Effects.
- Find Treatment Centers
Find an Authorized Treatment Center
By Using the Map Search Feature.
- Mechanism of Action
See How Gene Therapy Works.
Learn the Science Behind Treatment
- Prescribing Information
View Full Prescribing Information &
Indications And Usage.
- Treatment Journey
Learn More About Treatment And
See If It's Right for Your Patients
- HCP Resources
Download Treatment Brochures,
Billing Codes, and More.
- Safety and Side Effects
Search results
Results From The WOW.Com Content Network
A vaso-occlusive crisis is a common painful complication of sickle cell anemia in adolescents and adults. [1] [2] It is a form of sickle cell crisis.Sickle cell anemia – most common in those of African, Hispanic, and Mediterranean origin – leads to sickle cell crisis when the circulation of blood vessels is obstructed by sickled red blood cells, causing ischemic injuries.
Thalassemias are inherited blood disorders that result in abnormal hemoglobin. [7] Symptoms depend on the type of thalassemia and can vary from none to severe. [1] Often there is mild to severe anemia (low red blood cells or hemoglobin) as thalassemia can affect the production of red blood cells and also affect how long the red blood cells live. [1]
The discovery that hemoglobin F alleviated the symptoms of sickle cell disease occurred in 1948. Janet Watson observed that red blood cells from infants with the disease took longer to sickle and did not deform as much compared to their mother's cells, which carried the disease trait.
This treatment thus avoids the need for regular, costly blood transfusions. [13] [14] In December 2023, the FDA approved the first gene therapy in the US to treat patients with Sickle Cell Disease (SCD). The FDA approved two milestone treatments, Casgevy and Lyfgenia, representing the first cell-based gene therapies for the treatment of SCD. [57]
Autosplenectomy can occur in cases of sickle-cell disease where the misshapen cells block blood flow to the spleen, causing scarring and eventual atrophy of the organ. [2] Autosplenectomy is a rare condition that is linked to certain diseases but is not a common occurrence. It is also seen in systemic lupus erythematosus (SLE).
The company was founded as Genetix Pharmaceuticals in April 1992 by MIT faculty members Philippe Leboulch and Irving London. [7]In 2001, Walter Ogier was appointed chief executive officer of Genetix Pharmaceuticals, and the company was focused on the development of LentiglobinTM for the treatment of sickle cell disease and thalassemia major (beta-thalassemia), the two most globally prevalent ...
In persons with sickle cell disease, high levels of fetal hemoglobin as found in a newborn or as found abnormally in persons with hereditary persistence of fetal hemoglobin, the HbF causes the sickle cell disease to be less severe. In essence the HbF inhibits polymerization of HbS. A similar mechanism occurs with persons who have sickle cell trait.
Exa-cel is a drug therapy for the treatment of the rare blood disorders beta thalassemia and sickle cell disease developed jointly with Vertex Pharmaceuticals, [7] that received FDA approval for clinical use in December 2023. [5]
Ads
related to: sickle cell disease treatment