China's first Fabre disease special drug approved

　　Medicine Network December 24 - Sanofi China announced on December 24th that China's State Drug Administration has approved the long-term enzyme replacement therapy for patients diagnosed with Fabre's disease (alpha-semi-lactose assine A deficiency) for adults, children and adolescents 8 years and older The drug is the first approved drug in China to treat Fabre disease according to the introduction, Fabre disease (also known as Fabry disease) is a rare X-chromosome companion alsoxe storage disease, patients often appear like burning pain, short is sustained for several minutes, long for several days, sometimes repeated, serious when unable to normal life, and the kidneys, heart, brain, nerves and other organs causeserious damage to the disease Symptoms of this type of disease often occur in the patient's to adolescence
"Fabre disease is a very rare , although the pathological changes in the disease are typical, but due to the multiple organs, clinical manifestations are diverse, resulting in patients scattered in many departments, especially pediatrics, neurology, nephrology, rheumatology, digestive medicine, dermatology and , a rare disease scattered in so many departments, resulting in very difficult diagnosis, not only in China, other countries, often from diagnosis to diagnosis time of more than a decade Professor Yuan Yun, First Neurology, Peking University, said: "We have known and diagnosed Fabre disease for 20 years, although the long-term promotion of the diagnosis of the disease in China, but because of its rarity, its clinical diagnostic difficulties still exist, even if the patient swastorate, no special drugs before the diagnosis, many patients in the anxiety and pain of slow lying in despair, even some patients with the continuous development of the disease due to kidney failure or heart and brain damage and premature death." "
reported a statistically significant decrease in the total GL-3 in the kidneys, heart and skin of patients treated with Agaglyase beta in a placebo-controlled clinical trial (P?amp;lt;0.001) in patients treated with a placebo Another double-blind, placebo-controlled study showed that the rate of clinical events in patients treated with Agagase beta was significantly lower than in patients treated with placebo (incidence of kidney, heart, or cerebrovascular disease or death) Fabuzan has been listed in Europe and the United States and other countries and regions, the U.S Food and Regulatory Authority (FDA) is currently approved for the treatment of Fabre disease enzyme alternative treatment drugs (Complete)