Alpha-thalassemia–myelodysplastic syndrome (ATMDS)—also called acquired alpha-thalassemia with MDS, acquired HbH disease in MDS, or acquired α-thalassemia ...

Schindler disease type 3 is a very rare inherited condition. It happens when the body does not make enough of a lysosomal enzyme called ...

Alpha-N-acetylgalactosaminidase deficiency (often shortened to NAGA deficiency) is a very rare, inherited (passed from parents to children) condition. The body ...

Schindler disease type 2 is a very rare inherited (passed from parents) condition. It happens because the body does not make enough of an enzyme called ...

Kanzaki disease is a very rare inherited condition. It happens when the body does not have enough working of an enzyme called alpha-N-acetylgalactosaminidase ...

Adult-onset alpha-N-acetylgalactosaminidase deficiency is a very rare inherited (genetic) condition. In this condition, a body enzyme called ...

Alpha-N-acetylgalactosaminidase deficiency is a rare genetic condition in which the body does not make enough of an enzyme called ...

Alpha-N-acetylgalactosaminidase deficiency type 1 is a very rare, inherited (from both parents) disease of the body’s “recycling centers,” called lysosomes. In ...

Schindler disease is a very rare inherited (genetic) condition. People with this disorder do not have enough of an enzyme called ...

NAGA deficiency is a very rare inherited disease where the body does not make enough of an enzyme called alpha-N-acetylgalactosaminidase (often shortened to ...

Alpha-N-acetylgalactosaminidase—often shortened to alpha-NAGA—is a small worker-enzyme that lives inside the cell’s “recycling centers,” which are called ...

Alpha-N-acetylgalactosaminidase deficiency is a very rare inherited (runs in families) disease. It happens when the body does not make enough of one cleaning ...

Mannosidosis is a rare genetic disease. It belongs to a group called lysosomal storage disorders. Lysosomes are tiny recycling centers inside our cells. They ...

Alpha-mannosidosis is a rare, inherited disease. A child is born with it when both parents pass down a changed copy of a gene called MAN2B1. This gene makes an ...

Alpha-mannosidase deficiency—also called alpha-mannosidosis—is a rare inherited disease. It happens when the body does not make enough of an enzyme called ...

Dysostosis is a group of birth conditions that affect how certain bones form and join. The problem starts very early in pregnancy when the baby’s skeleton is ...