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For most of my life, my family has been advocating for therapies to help my twin brother. While the recent approval of a new drug should give us reason to celebrate, there is still more work to do.
My twin brother is 28 years old and has severe Hunter syndrome, also known as mucopolysaccharidosis (MPS) type II. Hunter syndrome is a rare condition caused by the deficiency of an enzyme needed to break down sugars called glycosaminoglycans (GAGs). Due to this enzyme deficiency, GAGs accumulate to toxic levels throughout the body, resulting in progressive multi-organ disease.
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