Original title: rare diseased new medicine In the first prescription patient, the first prescription patient can realize the domestic medication. Professor Deng Fang, director of the Nephrology Department of Children’s Hospital, Anhui Province, introduced X-chain low-phosphoric disease, innovative drug treatment. South Korea, in November 2021, used to treat X-chain low-phosphorite (XLH) and tumor bone softening (TIO) targeted therapeutic drug Lin Ping (Broosoui monk) in Anhui Children’s Hospital by kidney Professor Deng Fang, director of the internal medicine, opened the first prescription of Anhui.
The first place means that the innovative treatment plan landed in Anhui, which brought the breakthrough accurate treatment of low-phosphorus disease in the local rare disease. Hypophosphoric disease is a group of bone mineral disorders in renal phosphorus due to hereditary or acquisition, causing rare diseases characterized by hypospone characterized by hypospone, and is included in the "First Batch of Rare Disease Catalog" No. 51. Rare disease.
X chain low phosphoric disease, ie X chain low phosphot (XLH) is a kind of hypophosphorus disease, accounting for 80% of hereditary hypophostriction. The prevalence of XLH is approximately 1/60000 ~ 1/2000, and the number of patients is small, and the social attention is low. Professor Deng Fang, director of the Nephrology, Anhui Children’s Hospital, introduced that XLH is due to the excess of fibroblast growth factor 23 (FGF23) product due to PHEX gene mutation, has a high disability, teratogenicity. Before Brosso, there is no special medicine before the market, the patient can only use the treatment plan for oral phosphite to symptomatic treatment.
Deng Heng analyzed: "Even if it is used to supplement ‘phosphorus’ treatment, the blood phosphorus level in the patient is still less normal, and the skeleton of the deformation cannot be completely corrected, and it is difficult to improve the problem of growth and development in patients.
In addition, the phosphate preparation is taste, and the high frequency is used daily, and the patient compliance is poor.
The risk of complications such as renal calcium sedimentation, high urethropathy, and hyperotheral hyperparathyll hyperthyroide. Therefore, the listing of innovative special medicines of hypophosphorized disease is significant. "Lin Ping (Bromeo Eusi Anti-anti) can target the binding and inhibit the activity of FGF23, increase the renal rehabilitation phosphorus and serum active vitamin D level, and improve the level of serum phosphorus, is the only way to resolve XLH potential causes. Breakthrough therapy .
In a placebo, it was found that 94% of the subjects accepted a normal phosphorus level per month.
In recent years, with the importance of rare diseases, the development of rare diseases, the research and development of rare diseases, and the approval process has accelerated.
In 2019, Linping was included in the second batch of "clinical urgent needs of new drug list".
In January 2021, the Targeted Treatment of X Chain Hypoxiamia Thermal Burossoju was approved by the State Drug Administration (NMPA); March Bulo Eugene antibody expansion indication, NMPA approval Used for adult tumor bone softening; officially commercialized in July of the same year.
Deng Fang said: "The low-phosphorus disease not only causes the patient’s physical disability, but also brings a heavy economic and psychological burden to the patient’s family.
At present, clinically low-phosphoric diseases face diagnosis, mainly because clinicians have a lack of understanding of this rare disease. It is hoped that the community will work together to improve the knowledge of the disease and promote the knowledge of the overall diagnosis capacity. "(Han Suyuan) (Editor: Mouquet Yu, Zhu Hongxia) Sharing let more people see.