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    Home > Medical News > Latest Medical News > Boosting awareness of polysaccharide disease, first patient survival report officially released

    Boosting awareness of polysaccharide disease, first patient survival report officially released

    • Last Update: 2020-05-15
    • Source: Internet
    • Author: User
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    Guide: A number of senior associations to participate in the "International Polysaccharide Memory Care Day" officially beganAccording to the medical intelligence network was informed that on May 15, 2020, "International Sticky Polysaccharide Storage Care Day" came, by the Shanghai Rare Disease Prevention and Control Foundation, Shanghai Health and Health Development Research Center, Shanghai Medical Association Rare DiseaseS Branch and the Chinese Medical Science Branch of experts, scholars to participate in the "together, blooming life together Enzyme" - Mucopolysaccharidoses ("MPS") disease awareness campaign was officially launched to raise awareness of MPS disease among doctors, patients and the public through multidisciplinary seminars on MPS, as well as disease awareness and science for patients and the publicas the "International Polysaccharide Care Day" play, the organizers officially released "from the sticky polysaccharide aploty analysis of the survival status of rare disease patients in China" (hereinafter referred to as "report", click on thelinkview the report long picture), led by the above four units, Beijing Zhengyu polysaccharide rare disease care center strongly co-sponsored, and has been the North Sea Kangcheng, Million, Sanofi and other enterprises supportfor the first time to gain a comprehensive picture of the survival status of MPS patients
    MPS is a rare progressive disability, death x chain recessive genetic disease, which is now listed in the National List of Rare Diseases noThis report is the first study of the survival status of the MPS patient population in China and the largest sample size in china, with 180 patients participating, covering six dimensions: patient information, diagnostic experience, disease characteristics, treatment experience, quality of life, and patient burden, with the participation of clinical societies, research institutions and patient organizationsIt is reported that after diagnosis, in the domestic MPS patient organization signed up to 378 patients, of which about 84% of children, more than 200 patients are ready to registerthe report aims to understand the common problems of disease cognition, diagnosis and treatment and social security faced by the group of rare patients in China through multi-dimensional research on single disease, which will not only help promote MPS standardized diagnosis and treatment, help patients improve their quality of life and return to normal life, but also provide a reference for the establishment of China's rare disease security systemseveral difficult problems faced by the families of MPS patients
    the report found that the families of MPS patients in China are faced with several difficult problems that need urgent attention and solve:is the difficultof diagnosisMPS patients have an average of 6 visits before diagnosis, up to a maximum of 50The number of misdiagnosis can be up to 24 and the time can be up to 69 monthsProfessor Luo Xiaoping, vice-chairman of the Chinese Medical Science Branch, said, "The main reason for delayed diagnosis in MPS patients is the low awareness of clinical diseases, which is a common problem faced by many rare diseases." The fundamental solution to this problem lies in the establishment of MPS consultation norms, the organization of training and academic meetings, the establishment of a wide range of rare disease research collaboration groups and multidisciplinary diagnosis and treatment team, relatively concentrated diagnosis and treatment and two-way referral, and gradually achieve the goal of early detection of rare diseases, early diagnosis, treatment, management "
    second is the lack of treatment Due to the lack of effective specific drug treatment in The country, 41% of patients did not receive any treatment after diagnosis, 47% of patients were treated only for the disease, and even 8.95% of patients went overseas to receive treatment for specific drugs Professor Gu Xuefan, deputy director of the Shanghai Academy of Pediatric medicine and professor of pediatrics at Beijing Concord Hospital, points out that enzyme replacement therapy (ERT) is the specific standard treatment of MPS recommended by international guidelines I, II, IVa drugs in China has been or will be approved for market, drugs can improve the multi-organ system, improve patient growth and development, delay disease progression, prolong survival, improve health-related quality of life After receiving long-term specific treatment early, patients can marry and have children, study and work, and return to society third is the heavy care and the high cost reports that 60 per cent of patients have mobility difficulties, 54 per cent live on their own, 80 per cent have difficulty attending school, resulting in an average of 110 days of care and maintenance for their families and 63 per cent having to give up full-time work The average total expenditure on disease is $360,000, 1.61 times the annual household income, and 68% of households spend more than 40% of their household income on treatment, with an average of five months a year running out of money "MPS-specific treatment drugs are expensive and cannot be affordable for the average family, " said Jin Chunlin, director of the Shanghai Health and Health Development Research Center This is also a common problem encountered by many rare diseases, and the key to solving this problem is to establish a multi-level security system, explore local health insurance, financial support, social assistance, commercial insurance and enterprise multi-party cooperation mechanism, do not let patients because they can not afford to give up treatment dilemma "
    multi-party call: to improve China's rare disease diagnosis and protection system
    as one of the main sponsors of this disease awareness and patient survival report, Professor Li Dingguo, director of the Shanghai Foundation for rare disease prevention and control, said, "We launched this study to show from MPS as an entry point to show the common challenges facing the survival of rare disease patients in China, to help us further explore the establishment of China's rare disease diagnosis and treatment and protection system to address the problem of drug accessibility of rare diseases." We are also pleased to see that the country has begun to explore the establishment of a rare disease protection mechanism, some provinces and cities have also introduced a rare disease drug protection policy The future hopes to legislate to truly protect the 'last mile' of drugs available to patients with rare diseases "
    , Ms Zheng," said Ms Zheng,
    , head of the Beijing Zhengyu Sticky Polysaccharide Rare Disease Care Center, "Thanks to experts for systematically and scientifically combing the survival of MPS patients and reporting to the public the real situation our MPS patients suffer every day." It is hoped that the public and experts will continue to give more attention and support to the MPS patient group or even the rare disease patient group in China, 'Care starts with understanding', the completion of the investigation report is the first step to help patients out of the difficult situation, I hope that the survey can provide an effective basis for promoting the introduction of relevant MPS and rare disease policies "
    as one of the support units of this report, Dr Xue Qun, Chairman and CEO of Beihai Kangcheng Pharmaceutical Co., Ltd., said: "As a company based on the local rare disease new drug creation company, we not only provide patients with effective treatment programs for rare drugs, but also actively participate in promoting the construction of China's rare disease ecosystem, and work together with all parties to promote the establishment of relevant regulatory policies, especially how to solve the problems involved in drug accessibility, and ultimately let more rare patients and society benefit." "
    responsibility editor: penicillin related labels: Shanghai , rare diseases , polysaccharide memory , survival report 0
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