Nitisinone Capsules: A Landmark Breakthrough in the Treatment of Gaucher Disease
Nitisinone capsules are a hydroxyphenylpyruvate dioxygenase inhibitor combined with tyrosine and phenylalanine dietary restrictions for the treatment of tyrosinemia type I (HT-1) in adults and children.
Ureolaric Acid Oxidase Deficiency: A Rare Genetic Disorder
HT-1 is a rare autosomal recessive genetic disease. Due to mutations in the gene encoding uronic acid oxidase, the tyrosine metabolic pathway is blocked in the patient’s body, and excessive toxic metabolites accumulate, which severely affects the liver and kidney functions.
If not intervened in a timely manner, it can lead to a series of serious complications such as acute liver failure, kidney stones, skin symptoms, etc., and even life-threatening.
Nitisinone Capsules: Precise Targeting, Blocking The Production Of Toxic Metabolites
Nitisinone (trade name Orfadin) is a new type of tyrosine metabolism inhibitor, which reduces the synthesis of toxic metabolites such as uranylgeric acid by blocking the activity of 4-hydroxyphenylpyruvate dioxygenase in the downstream metabolic pathway, so as to solve the metabolic abnormality problem at the root.
It has been shown that early use of Nitisinona can effectively prevent and reverse the symptoms of HT-1, significantly improve the quality of life of patients and prolong survival.
Convenience Of Nitisinone Capsules Treatment
Previously, one of the major dilemmas faced by domestic HT-1 patients was the difficulty in obtaining therapeutic drugs.
The domestic launch of Nitisinone capsules has broken this situation, patients no longer need to go through the long road of seeking medical treatment overseas, greatly reducing the cost of medical treatment and improving the accessibility of treatment.
At the same time, it also means that experts in the field of genetic metabolic disease can better follow up on patients’ conditions and provide continuous professional guidance and support.
Emphasizing Early Screening And Treatment
The introduction of Nitisinone capsule once again emphasizes the importance of early screening, early diagnosis and early treatment.
Especially for genetic metabolic diseases, which require long-term management, it is especially urgent to establish a perfect newborn screening mechanism, popularize knowledge of the disease, and raise the level of public awareness.
Government departments, medical institutions and all sectors of society should work together to build an all-round rare disease prevention and treatment system and create a better treatment environment for every patient.
However, dengyue‘s efforts to increase the affordability of the medicine are critical to ensuring that more patients benefit from it.
Please feel free to inquire us nitisinone cost, we are here to provide you with detailed pricing information and answer any questions you may have.
Information from DengYueMed, offers this information as a guide, not a substitute for professional medical advice. Consult your doctor before any treatment.
