Anna Ficcadenti

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Mucopolysaccharidosis type I is an autosomal recessive disorder caused by deficiency of α-L-iduronidase and characterized by a progressive course with multisystem involvement. Clinically, Mucopolysaccharidosis type I is classified into two forms: severe (Hurler syndrome), which presents in infancy and is characterized by rapid progressive neurological(More)
Progress in medical and scientific research has increased the chances of survival for young patients with congenital diseases, children who, in the past, would not have had any chance of survival. Nowadays, congenital diseases can be treated with appropriate replacement therapies. These treatments can be difficult to administer in young patients because of(More)
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