3. MPS Disease Treatment Options Overview


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In relation to treatment, it of utmost importance to consider symptomatic treatment in patients, all organs and organ systems can be affected, so pediatricians should think about how to deal with all these signs and symptoms in the different organs and organ systems. 

  • In addition, there are disease modifying treatments for a number of these disorders and this involves hematopoietic stem cell transplantation, formerly bone marrow transplantation nowadays often umbilical cord stem cell transplantation for the severe phenotype of MPS I, the Hurler phenotype, and in a number of the others enzyme replacement therapy, intravenous infusion with the deficient enzyme, is the treatment option of choice at this moment. However, early recognition is essential, the success of treatment depends on the early initiation of treatment.
  • With regards to patients with Hunter Syndrome (MPS II) it essential that the physician is aware of the multi-systemic issues (e.g. nervous, respiratory and musculoskeletal systems) surrounding the patient, therefore a multidisciplinary team approach is required. Due to the broad nature of the clinical symptoms, often the involvement of specialists such as anesthetists, cardiologists, neurologists, neurosurgeons, pediatric surgeons, ophthalmologists, orthopedic surgeons, pulmonologists and otorhinolaryngologists is required. 

 

 

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