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Genetic and Rare Diseases Information Center (GARD)

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Hereditary spherocytosis

Other Names for this Disease
  • Congenital spherocytic hemolytic anemia
  • Congenital spherocytosis
  • Spherocytic anemia
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Your Question

Can Soliris be used to cure hereditary spherocytosis?

Our Answer

We have identified the following information that we hope you find helpful. If you still have questions, please contact us.

What is Soliris?

Soliris is a drug that has been FDA approved to treat paroxysmal nocturnal hemoglobinuria (PNH). PNH causes the early death and impaired production of blood cells (red blood cells, white blood cells, and platelets). This early destruction of blood cells occurs because the affected cells are missing two important proteins, making them vulnerable to destruction by a part of our immune system called the complement system.[1] Soliris works by blocking the complement system. You can learn more about Soliris by visiting the Web page on this topic. 
Last updated: 5/3/2011

Can Soliris be used to treat hereditary spherocytosis?

Currently, we are not aware of any benefits of Soliris for treating hereditary spherocytosis. The destruction of red blood cells in this condition is a result of mishapen and fragile cell membranes.[2]
Last updated: 5/3/2011

How can I learn about clinical trials and research studies involving new treatments for hereditary spherocytosis?

To learn more about research that is currently being conducted on hereditary spherocytosis, you can view our Clinical Trials & Research resource tab.
Last updated: 5/3/2011