Related Conditions and Treatments
Acute Lymphoblastic Leukemia (ALL)
In the form of leukemia known as acute lymphoblastic leukemia (ALL), a group of white blood cells called lymphocytes is affected.
Adrenoleukodystrophy (ALD) is a rare genetic condition that causes the buildup of very long chain fatty acids (VLCFAs) in the brain.
Chronic Granulomatous Disease (CGD)
Chronic granulomatous disease (CGD) is an inherited immune system disorder that occurs when a type of white blood cell does not work properly.
Gene therapy is a technique used in an effort to treat or prevent disease.
Hemophilia is a bleeding disorder that slows the body’s ability for form blood clots.
Ornithine Transcarbamylase (OTC) Deficiency
OTC deficiency is caused by mutations in the gene encoding the ornithine transcarbamylase (OTC) enzyme.
Severe Combined Immunodeficiency (SCID)
Severe combined immunodeficiency (SCID), often called “bubble boy disease,” is very rare, genetic disorder.
Sickle Cell Disease
Sickle cell disease is an inherited blood disorder where cells cluster together, making it difficult for them to move through small blood vessels, thus denying oxygen to the blood.
Spinal Muscular Atrophy (SMA)
Spinal muscular atrophy is when muscles throughout the body are weakened because nerve cells in the spinal cord and brainstem do not work properly.
Thalassemia is an inherited blood disorder that causes the body to produce less hemoglobin than normal.
Wiskott-Aldrich syndrome is a rare genetic immunodeficiency that keeps a child's immune system from functioning properly.