Intracerebroventricular Enzyme Replacement Therapy in Patients with Neuropathic Form of Mucopolysaccharidosis Type II: to Help Practicing Physician

• Main Authors: Nataliya V. Zhurkova (Author), Nato D. Vashakmadze (Author), Georgiy A. Karkashadze (Author), Elena V. Kaytukova (Author)
• Format: Book
• Published: "Paediatrician" Publishers LLC, 2024-09-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Mucopolysaccharidosis type II (Hunter syndrome, MPS II) is a rare hereditary disease from the group of hereditary metabolic diseases. There are neuropathic and non-neuropathic forms of this disease. The neuropathic form is most common and leads to severe cognitive impairment and progressive damage o...