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.
CASGEVY™ Gene Therapy for Sickle Cell Disease and Beta Thalassemia
Treatment
CASGEVY (exagamglogene autotemcel) is a gene editing therapy for those 12 or up with sickle cell disease or beta thalassemia.
Learn more about CASGEVY™ Gene Therapy for Sickle Cell Disease and Beta Thalassemia
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.
Elevidys
Treatment
Elevidys® (delandistrogene moxeparvovec-rokl) is a gene therapy for Duchenne muscular dystrophy in boys aged 4 and 5.
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.
Inherited Retinal Disorders
Condition
An inherited retinal disorder (IRD) alters the structure and function of the retina and impairs vision.
LYFGENIA™ Gene Therapy for Sickle Cell Disease
Treatment
LYFGENIA is a gene therapy treatment for people 12 or older with sickle cell disease and a history of pain crises.
Learn more about LYFGENIA™ Gene Therapy for Sickle Cell Disease
Luxturna
Treatment
Luxturna (voretigene neparvove) is a gene therapy treatment for inherited retinal disorders caused by RPE65 mutations.
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.
Zolgensma
Treatment
Zolgensma® (onasemnogene abeparvovec-xioi) is a gene therapy for spinal muscular atrophy (SMA) in children under 2.