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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.

Heterotaxy is a rare condition where many organs in the body can be formed abnormally, in the wrong position, or even missing.

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

A transnasal endoscopy is a minimally invasive procedure used to examine the upper digestive tract.

Stridor is a high-pitched sound that is usually heard best when your child breathes in (known as “inspiration”). It’s usually caused by an obstruction or narrowing in your child's upper...

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

22q11.2 deletion syndrome is a genetic condition that causes a combination of medical problems. These problems may include heart defects, cleft palate, speech or feeding problems, difficulty fighting infections, low...