How to treat rare diseases? The company has developed a new generation of pituitary hormone pump, which will effectively treat "gender free" Kalman syndrome

"As a man, I have no Adam's apple, no voice change, or even secondary sexual characteristics Walking on the road, some call me sissy, some call me little sister " Before the age of 23, this kind of voice from gender misunderstanding, like a net, tighter and tighter, makes pan Longfei hard to breathe, or even once fell into inferiority What's his matter? People around you often ask questions In an interview with the arterial network, pan Longfei, 31, told reporters that he was suffering from a rare disease called Kalman syndrome Kalman's syndrome is a hypogonadotropic hypogonadism with anosmia or hyposmia It is a disease with clinical and genetic heterogeneity Generally speaking, patients with Kalman's syndrome "can't smell the fragrance, have no sexual desire, and can't bear" According to statistics, the incidence rate of male Calman syndrome is about 1/8000, and the incidence rate of female is about 1/40000 On May 11, 2018, five departments including the national health and Health Commission jointly formulated the catalogue of the first batch of rare diseases, in which Kalman syndrome was included What is the cause of Kalman syndrome? What specific dilemmas are patients facing at present? Are there innovative therapies that can help them With these questions, the arterial network interviewed pan Longfei, a patient with Kalman syndrome, and kailian medical, a company dedicated to developing innovative therapies for Kalman syndrome, to try to make some answers On the edge of the world, they have few medicines, are hard to cure and almost no medical insurance There are 7000 kinds of rare diseases around the world But less than 10% of rare diseases have effective drug treatment, and the rest 90% are still waiting for innovation in medical science and technology This data means that, in terms of treatment, the vast majority of patients with rare diseases can only be "resigned" Pan Longfei is the founder of "old K's home" which is a public welfare organization for patients with Kalman's syndrome In 2012, "old K's home" was established Since its establishment 7 years ago, there have been more than 3000 registered patients "Many of the patients I met were very self abased and depressed It's hard for them to accept that they don't even know they have Kalman syndrome " Pan Longfei said Pan Longfei introduced that there are 200000 patients with Kalman syndrome in China Men with Kalman's syndrome have no pubic hair, armpit hair, beard, laryngeal node, no voice, and immature external genitalia; women with Kalman's syndrome have no menarche, no pubic hair, armpit hair, undeveloped breast, internal genitalia, and immature ovary and uterus In clinic, Kalman syndrome is similar to most rare diseases, it can't be cured and can only be treated conservatively by drugs At present, there are three mainstream treatment programs: first, androgen replacement, no fertility requirements can be treated after the age of 14, the goal is to promote the development of male secondary sexual signs; second, to promote subcutaneous injection of adenohormone, the goal is to restore fertility; 3、 GnRH pulse pump, simulating the physiological GnRH secretion, promotes the secretion of gonadotropin in the anterior pituitary, promotes the development of testis and ovary, and restores fertility In 2014, due to the discontinuation of the drug "fluroxypsin for injection" used for Kalman's syndrome, the old K's family initiated the action "I am old K, I need fluroxypsin for injection", and finally got the attention of the competent government departments and the society, which solved their drug use problems well (Photo by interviewee: old K's house walks in vain) "for marginalized us, in addition to the huge psychological burden, lack of medicine, difficult to cure and almost no medical insurance are the multiple difficulties we are facing and difficult to overcome." Pan Longfei Because of the small number of Calman syndrome, the limited profit space, the production of drugs is very few; moreover, its incidence rate is low, the disease is special, resulting in less medical resources The vast majority of patients are faced with the situation that they can't be diagnosed or treated correctly after diagnosis Only going to hospitals in big cities such as beishangguang can they get timely and accurate treatment And the huge economic burden brought by the treatment in different places also makes the patients shrink away Diagnosis and treatment of these problems is undoubtedly a mountain in front of young patients As time goes on, "marriage" and "childbearing" will become new life problems Is there any way to help such a group of people? According to Huang Xiaomin, founder of kailian medical, the original intention of the new generation of pituitary hormone pump launched by kailian medical is to solve these problems well and bring good news for Kalman patients Ten years of innovative research and development of pituitary hormone pump, "artificial" hypothalamus treatment of Kalman patients, some say, rare disease is a struggle history around a small number of people Founded in 2014, kailian medical is a company dedicated to the R & D and production of innovative micro drug infusion equipment The product line covers new insulin pump, hormone pump, electric insulin pen, sticking pump and other innovative products Among them, its independently developed new generation of pituitary hormone pump is in the leading position in China What is the pituitary hormone pump developed by kailian? "In fact, the essence of Kalman's syndrome is hypogonadotropic hypogonadism (IHH) The problem of gonads is closely related to the hypothalamus pituitary gonad axis of the endocrine system Said Huang Xiaomin He introduced that the second generation of pituitary hormone pump independently developed by kailian can effectively stimulate the pituitary to secrete gonadotropins, and then promote the gonadal development, secretion of sex hormones and the generation of sperm or follicles through the way of subcutaneous injection of gonadotropin releasing hormone (GnRH) analogues by means of a micro input device controlled by artificial intelligence to simulate the physiological pulse secretion mode of the hypothalamus Yes, let the patient gain fertility (the picture shows kailian pituitary hormone pump) according to the 2017 version of "gonadotropin releasing hormone (GnRH) pulse treatment expert consensus", this treatment method is applicable to all patients with abnormal GnRH pulse secretion, intact pituitary gonad reserve function and fertility intention For example: interruption of pituitary stalk, Kalman syndrome, IHH, idiopathic central amenorrhea, non obese PCOS and other diseases Huang Xiaomin said that kailian pituitary hormone pump has rich functions It is equipped with OLED color large screen with clear display, convenient operation of capacitive touch screen, safe rechargeable battery, dose segmented adjustment suitable for different personalized treatment schemes, Bluetooth transmission of treatment information to mobile phone and upload to cloud server, realizing the sharing and storage of treatment data For patients, it is very convenient to use and fully realize personalized treatment Why dabble in rare diseases, research and development of pituitary hormone pump? Huang Xiaomin tells the story of his association with rare diseases and old K's In 2009, he worked as vice president of minimally invasive medicine, managing the development of endocrine and metabolic disease treatment products "At that time, we cooperated with Shanghai Ruijin Hospital, and during the clinical research, we found that many men in their twenties were very tall and beautiful, but they had no secondary sexual characteristics and could not have children." Said Huang Xiaomin "Later, we learned from experts at Ruijin hospital that these are all patients with Kalman syndrome Their living conditions are generally not very good, and they are a less concerned group I was wondering if I could find an innovative therapy to help them Huang Xiaomin told the arterial network After continuous research and development tests with clinical experts of Ruijin Hospital, the first generation of pituitary hormone pump products was finally born At that time, a 22-year-old male patient found a clinical experimental group, hoping to try the research and development results "The patient came to us and said that if he could not have a child, his wife might divorce him, leading to the breakdown of the family Whether the result is good or bad, he wants to try our treatment plan " Huang Xiaomin said Less than a year after treatment, the man's second sexual characteristic symptoms improved significantly "This is the first patient to have a successful birth He also sent an email to express his thanks and attached a picture of his child Our products can help him find family happiness, and our hearts are also very happy " Said Huang Xiaomin In 2014, Huang Xiaomin and Junlian capital jointly founded kailian medical to build an incubation platform for innovative medical equipment Two years later, kailian medical transformation focused on chronic disease and rare disease management, and deeply engaged in the field of Endocrinology In 2009-2019, 10 years later, Huang Xiaomin redefined and upgraded the pituitary hormone pump product based on the research and development of the original generation He said that whether it is a generation of products or upgraded new generation of pituitary hormone pump, it is currently in a leading position in China The characteristics of the new generation of pituitary hormone pump just on the market are shown in three aspects: first, technological breakthrough The first generation of product is the product transformed on the ordinary insulin pump The new generation of pituitary hormone pump is a specially developed pulse infusion mechanism, which truly realizes the function of artificial hypothalamus without side effects and has outstanding effect 2、 Better user experience Compared with the first generation of products, the new generation of pituitary hormone pump has made more optimization, such as the use of color LCD screen, equipped with Bluetooth function and disease diagnosis and treatment software platform, which can real-time monitor the situation of patients and facilitate doctors' follow-up 3、 Lower the price For patients with Kalman, the treatment cycle is long and needs continuous treatment Compared with the first generation products, we will consider to reduce the price of products, reduce the cost of treatment, and better serve patients At present, the new generation of pituitary hormone pump has been approved by CFDA We have also reached cooperation with many well-known hospitals, such as Beijing Union Medical College Hospital, General Hospital of the people's Liberation Army, Shandong Provincial Hospital, Beijing Chaoyang Hospital, etc Many patients indicated that the effect was good and the symptoms were greatly improved As a rare disease requiring long-term treatment, the treatment management of patients is very important for doctors and patients At the beginning of its establishment, kailian Medical Co., Ltd has made early layout in product intelligence and cloud data exchange It has jointly developed app platform and cloud data exchange platform for rare diseases with partners It hopes to provide long-term services for patients with chronic diseases and rare diseases, establish communication channels between doctors and patients, and form a care network, so that patients' treatment can get doctors' attention at any time In the future, kailian will cooperate with all drug infusion equipment to provide a more complete integrated solution for drug treatment of rare diseases, give patients and doctors more choices, and provide valuable information for the research work of rare disease treatment "We can't change the disease, but we can change the life of the patient." Said Huang Xiaomin He said that with the strong technical team of kailian medical, in the future, it will also make layout on rare diseases, develop more practical products, and help patients with rare diseases rebuild their confidence in life Penicillin is the reprinted content of yaozhi.com, and the copyright belongs to the original author The purpose of reprinting is to transmit more information, which does not represent the view of this platform If the content of the work, copyright and other issues are involved, please contact our website message, we will delete the content in the first time.