What is Alpha Mannosidosis
Giacomo Chiesi, Head of Chiesi Global Rare Diseases, gives an overview of alpha mannosidosis.
Alpha mannosidosis is a multisystemic, lysosomal disorder caused by a deficiency in the enzyme alpha-D-mannosidase that leads to an abnormal accumulation of glycoproteins that can damage various tissues and organs.
The symptoms and severity of alpha mannosidosis are highly variable.
Symptoms may include distinctive facial features (e.g., prominent forehead and jaw, and a flattened nose), skeletal abnormalities, hearing loss, intellectual disability, and dysfunction of the immune system.
Recently, velmanase alfa was approved by the U.S. Food and Drug Administration (FDA) for the treatment of non-central nervous system manifestations of alpha mannosidosis adult and pediatric patients.
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