Breakthrough Therapies Transforming Mucopolysaccharidosis Type I: A New Era of Hope
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...
0 0 Comentários 0 Compartilhamentos