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Von Willebrand Disease (VWD) What is VWD? Von Willebrand disease (VWD) is an inherited disorder that affects the blood's ability to clot. What causes VWD? VWD is almost always inherited. This means it is passed from parents to children. People with the disorder may have low

Diamond Blackfan Anemia (DBA) in Children What is DBA in children? Diamond Blackfan anemia (DBA) is a rare blood disorder. Children with DBA don't make enough red blood cells. These cells carry oxygen to all other cells in the body. Blood cells are made in the bone marrow, the

Short Bowel Syndrome in Children What is short bowel syndrome in children? Short bowel syndrome is when the body has trouble absorbing nutrients from food because of a problem with the small intestine. It often happens to children who have had a large part of their small

Langerhans Cell Histiocytosis in Children What is Langerhans cell histiocytosis in children? Langerhans cell histiocytosis is a rare disorder. It occurs when there are very high levels of a type of immune cell called a Langerhans cell. In the past, this disorder was thought to be

Tongue-Tie (Ankyloglossia) in Children What is tongue-tie in children? Tongue-tie (ankyloglossia) is a problem with the tongue that is present from birth. It causes speech and eating problems in some children. The lingual frenulum of the tongue is a small fold of tissue that

Duchenne Muscular Dystrophy in Children What is Duchenne muscular dystrophy in children? Duchenne muscular dystrophy (DMD) is a genetic condition that weakens your child’s muscles. It is the most common muscular dystrophy, a kind of inherited muscle disease. DMD causes weakness

Astrocytoma in Children What is an astrocytoma in children? An astrocytoma is a type of brain tumor. A brain tumor is an abnormal growth of tissue in the brain. The brain is part of the central nervous system (CNS). The CNS also includes the spinal cord. The main parts of the

Emery-Dreifuss Muscular Dystrophy in Children What is Emery-Dreifuss muscular dystrophy in children? Emery-Dreifuss muscular dystrophy (EDMD) is one of the muscular dystrophies. These are a group of rare, inherited muscle diseases (myopathies). EDMD causes weakness in your child

Facioscapulohumeral Muscular Dystrophy in Children What is FSHD in children? Facioscapulohumeral muscular dystrophy (FSHD) is a rare genetic muscle disease that affects the muscles of your child’s face, shoulders, upper arms, and lower legs. These muscles weaken and shrink

Congenital Liver Defects What are congenital liver defects? Congenital liver defects are liver disorders that are present at birth. They are rare. These liver disorders usually block the bile ducts. This affects the flow of bile. Bile is a fluid made in the liver. It helps with