How to treat mucopolysaccharide storage disease?

1.Hematopoietic stem cell transplantation: This is a specific treatment for mucopolysaccharide storage disease, which originated in the 1980s. The biggest disadvantage of hematopoietic stem cell transplantation is the presence of a high mortality rate, probably around 70%. The current consensus is that in severe patients with type I, HSCT before the age of 2 years can alter the natural course of the disease, promote height gain, and improve multiple organ storage symptoms, especially in the nervous system, but not in the heart valves that have already occurred. Bone marrow transplantation is also effective in type VI patients, but over time, HSCs may be gradually eliminated. In the past, it was thought that hematopoietic stem cell transplantation had no effect on neurological involvement in type II, but recent Japanese data show that bone marrow transplantation before the age of 3 is still effective. 2. Enzyme replacement therapy: This is also a specific treatment for mucopolysaccharide storage disease, which is provided by intravenous infusion route. The enzyme deficiency is obtained by bioengineering method. The advantage of enzyme replacement therapy is its safety. Currently, enzyme replacement therapy is available for Type I, Type II and Type VI. The enzyme for type IVA is currently in the clinical drug trial stage. Enzyme replacement therapy is preferred for patients with mild type VI and type I. Enzyme replacement therapy should also be administered during the perioperative period for HSCT in severe patients. Enzyme replacement therapy is very expensive. 3. Symptomatic supportive therapy: such as rehabilitation, heart valve replacement, hernia repair, cochlear implant, and cornea transplantation to improve the patient’s quality of life.