Related Conditions and Treatments
Acute Lymphoblastic Leukemia (ALL)
Condition
In the form of leukemia known as acute lymphoblastic leukemia (ALL), a group of white blood cells called lymphocytes is affected.
Adrenoleukodystrophy (ALD)
Condition
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)
Condition
Chronic granulomatous disease (CGD) is an inherited immune system disorder that occurs when a type of white blood cell does not work properly.
Duchenne Muscular Dystrophy
Condition
Duchenne muscular dystrophy (DMD) is the most common and most severe type of muscular dystrophy.
Gene Therapy
Treatment
Gene therapy is a technique used in an effort to treat or prevent disease.
Hemophilia
Condition
Hemophilia is a bleeding disorder that slows the body’s ability for form blood clots.
Ornithine Transcarbamylase (OTC) Deficiency
Condition
OTC deficiency is caused by mutations in the gene encoding the ornithine transcarbamylase (OTC) enzyme.
Learn more about Ornithine Transcarbamylase (OTC) Deficiency
Severe Combined Immunodeficiency (SCID)
Condition
Severe combined immunodeficiency (SCID), often called “bubble boy disease,” is very rare, genetic disorder.
Sickle Cell Disease
Condition
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)
Condition
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
Condition
Thalassemia is an inherited blood disorder that causes the body to produce less hemoglobin than normal.
Wiskott-Aldrich Syndrome
Condition
Wiskott-Aldrich syndrome is a rare genetic immunodeficiency that keeps a child's immune system from functioning properly.