Mucopolysaccharidosis Type I (MPS I) is a rare genetic disorder that disrupts the body's ability to break down glycosaminoglycans (GAGs), leading to severe organ and tissue damage. While traditional treatments like enzyme replacement therapy (ERT) have provided significant benefits, the search for more effective and long-lasting therapies continues. In this article, we will explore...

    Mucopolysaccharidosis Type I (MPS I) is a rare genetic disorder that disrupts the body’s ability to break down GAGs, leading to serious health issues like organ enlargement, skeletal abnormalities, and neurological decline. Although MPS I is considered a challenging condition to treat, advances in medical science have led to significant improvements in care. Traditional...

The journey of treating Mucopolysaccharidosis Type I has evolved from a time when families received devastating diagnoses with little hope for intervention to an era where multiple therapeutic options offer genuine prospects for improved outcomes. This genetic disorder, resulting from mutations in the IDUA gene that encodes alpha-L-iduronidase, creates a complex web of clinical manifestations...

    Mucopolysaccharidosis Type I (MPS I) represents one of the most challenging inherited metabolic disorders, affecting approximately 1 in 100,000 births worldwide. This rare genetic condition occurs when the body lacks sufficient alpha-L-iduronidase enzyme, leading to the harmful accumulation of glycosaminoglycans in cells throughout the body. While current treatments have...