A newly developed medicine to treat Mucopolysaccharidosis II (MPS II) disease — also called Hunter Syndrome — will benefit nine patients in Taiwan, Tsai Chiung-wei, founder of the Taiwan Mucopolysaccharidosis Association said yesterday.
Tsai said the new medicine, named Elaprase, is the first product to treat the rare inherited disease caused by deficiency of a specific enzyme to metabolize mucopolysaccharide in the human body, which leaves the patient with severe organ damage.
MPS II has more patients than other types of MPS disease. Taiwan has 49 patients who have MPS II, but only nine of them — aged from 5-22 — are able to be treated with Elaprase. The other patients are either too young or have brain damage that is too advanced to be treated by the medicine.
Medicines for MPS I and VI developed several years ago have helped five and seven Taiwanese patients, respectively, Tsai added.
Lo Yan, a 13-year-old student with MPS II, is the first patient to receive Elaprase treatment in Taiwan. His father said they have been waiting for three years for the medicine since Japan announced the start of clinical tests.
Tsai said Taiwan is the first country in Asia to cover the cost of the medicine with national health insurance. A patient like Lo would have to pay NT$1.6 million (US$48,341) per month if it were not covered by health insurance.
Lin Hsuan-pei, director of pediatric genetics at Mackay Memorial Hospital, said the medicine is a synthetic enzyme that stops accumulation of mucopolysaccharide in the body and helps extend the patient’s life.