Related Conditions & Treatments | Boston Children's Hospital

Condition

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.

Find a Doctor

Condition

Adrenoleukodystrophy (ALD)

Adrenoleukodystrophy (ALD) is a rare genetic condition that causes the buildup of very long chain fatty acids (VLCFAs) in the brain.

Find a Doctor

Treatment

CASGEVY

CASGEVY (exagamglogene autotemcel) is a gene editing therapy for those 12 or up with sickle cell disease or beta thalassemia.

Find a Doctor

Condition

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.

Find a Doctor

Condition

Duchenne Muscular Dystrophy

Duchenne muscular dystrophy (DMD) is the most common and most severe type of muscular dystrophy.

Find a Doctor

Treatment

Elevidys

Elevidys® (delandistrogene moxeparvovec-rokl) is a gene therapy for Duchenne muscular dystrophy in boys aged 4 and 5.

Find a Doctor

Treatment

Gene Therapy

Gene therapy is a technique used in an effort to treat or prevent disease.

Find a Doctor

Condition

Hemophilia

Hemophilia is a bleeding disorder that slows the body’s ability for form blood clots.

Find a Doctor

Condition

Inherited Retinal Disorders

An inherited retinal disorder (IRD) alters the structure and function of the retina and impairs vision.

Find a Doctor

Treatment

KEBILIDI

KEBILIDI™ (eladocagene exuparvovec-tneq) is a gene therapy for AADC deficiency. It enables brain cells to make a working AADC protein needed for motor function.

Find a Doctor

Treatment

Luxturna

Luxturna (voretigene neparvove) is a gene therapy treatment for inherited retinal disorders caused by RPE65 mutations.

Find a Doctor

Treatment

LYFGENIA

LYFGENIA is a gene therapy treatment for people 12 or older with sickle cell disease and a history of pain crises.

Find a Doctor

Condition

Ornithine Transcarbamylase Deficiency

OTC deficiency is caused by mutations in the gene encoding the ornithine transcarbamylase (OTC) enzyme.

Find a Doctor

Treatment

ROCTAVIAN

ROCTAVIAN (valoctocogene roxaparvovec-rvox) is a one-time gene therapy treatment for severe hemophilia A.

Find a Doctor

Condition

Severe Combined Immunodeficiency

Severe combined immunodeficiency (SCID), often called “bubble boy disease,” is very rare, genetic disorder.

Find a Doctor

Condition

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.

Find a Doctor

Treatment

Skysona

SKYSONA (elivaldogene autotemcel) is a one-time gene therapy for boys ages 4 to 17 with early X-linked cerebral adrenoleukodystrophy (CALD).

Find a Doctor

Condition

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.

Find a Doctor

Condition

Thalassemia

Thalassemia is an inherited blood disorder that causes the body to produce less hemoglobin than normal.

Find a Doctor

Condition

Wiskott-Aldrich Syndrome

Wiskott-Aldrich syndrome is a rare genetic immunodeficiency that keeps a child's immune system from functioning properly.

Find a Doctor

Treatment

Zolgensma

Zolgensma® (onasemnogene abeparvovec-xioi) is a gene therapy for spinal muscular atrophy (SMA) in children under 2.

Find a Doctor

Treatment

ZYNTEGLO

ZYNTEGLO™, also called beti-cel (betibeglogene autotemcel), is a gene therapy for children and adults with beta-thalassemia who require regular transfusions of red blood cells.

Find a Doctor