Disease only transmitted to females
Correct Answer: Hunter's disease
Description: Hunter disease (MPS II) is an X-linked disorder due to a deficiency in iduronate sulfate sulfatase and has manifestations similar to those ofMPS I, including neurologic degeneration. There is no corneal clouding or other eye diseases. Hematopoietic stem cell transplantation has not been successful in treating CNS disease associated with MPS II. The FDA and the European Medicines Agency (EMA) have approved enzyme therapy for the visceral manifestations of MPS II. References: Harrison's Principles of Internal Medicine; 19th edition; CHAPTER 432e; Lysosomal Storage Diseases
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