“Rare diseases” are a global public health problem. including Thailand Previously, it was a group of diseases that were difficult to detect due to lack of appropriate tools. But overall, there are not many patients. At present, there are approximately 400 million rare disease patients worldwide, of which more than 3.5 million are patients in Thailand, but only 20,000 of them are properly diagnosed and treated.
Takeda Thailand as a global biopharmaceutical company Continue to raise awareness to promote the standard of care for patients with rare diseases at the event. “Embrace the Differences, Unite for Rare Diseases” Tell an inspirational story from a patient with Gochere’s disease. one of the rare diseases Through the story exhibition “Gocher and the Magical Power of Secrets” and hear about advances in medical innovation that will improve patient care and treatment. for better quality of life of patients and families which is the hope of patients with thousands of rare diseases while one patient’s voice is an important link to other patients. And spread to the wider society to recognize their value and needs. In addition, within the event, there is also a flower arrangement workshop that will let you know that the flowers around you hide good meanings. to help create positive energy and relaxation for both patients and caregivers.
Mr. Peter Streibel, Managing Director of Takeda (Thailand) Co., Ltd. said, “Takeda is committed to its mission of pursuing better health and a brighter future for people around the world. through the development of innovative treatments that will promote a better quality of life The rare disease is one of the groups of diseases that we focus on. We work with public health agencies. medical organization Including a group of patient representatives to jointly support the creation of opportunities and equality for patients to access treatment and lead to a better future for the rare disease community.”
Prof. Dr. Worasak Choti Lersak Director of the Center of Excellence in Genetics Chulalongkorn University Said, “Each disease in the rare disease group has a small number of patients. But since there are more than 7,000 diseases in this group, there are many patients in total. This makes diagnosing rare diseases even more difficult. but nowadays There is a state-of-the-art tool called “Next Generation Sequencing” or “NGS”, which is a technology to transcribe the entire genome in a short time. This makes it possible to detect genetic abnormalities that are the main cause of rare diseases. Doctors are therefore able to make a quick diagnosis. leading to timely treatment It gives the patient a chance to survive and have a better quality of life. It often turns away from having a lifelong caregiver. become a power of society.”
while Boon Puttipongthanachot President of LSD Genetic Diseases Foundation A father who takes care of a child with Gocheer disease Said, “Nong Fai had Gosher disease since birth. At that time, the disease was very new to us. From day one, I only knew that I had to do everything I could to help my child survive. Moreover, the treatment process is complicated and expensive. It’s a once in a lifetime challenge. But we got through it. therefore looking back on other patients who had to face this difficulty, what would he do? This was the inspiration for the founding of the Better Genetic LSD Foundation in Thailand. Collected patients with Gocheer disease and other rare diseases among LSD genetics. come together Ready to push for the rights that the government takes care of Finally, from the efforts of all of us. Gosher Drug Type 1 is listed on the National List of Essential Medicines. Help finance and access treatment for patients and caregivers. We will continue to work with all sectors to drive rare diseases to be treated equally in the future.”
Rare diseases are genetic diseases that are classified as diseases that have a low incidence and a small number of cases relative to the population. Relevant research and medical data are limited. Thus, there are few studies about these diseases. The number of rare diseases is 6,000-8,000 such as hemophilia, Gaucher, Fabry, and hereditary angioedema (HAE). Genetic inheritance caused by a recessive gene inherited by a patient from both parents. Lysosomal storage disorder (LSD) causes physical abnormalities from the accumulation of substances in the lysosomes (Lysosomal Storage Disorder or LSD). Gocher’s disease is divided into 3 types: type 1 has no neurological symptoms; type 2 has acute neurological symptoms. Patients die rapidly from neurological complications and type 3 patients have neurological symptoms. There will be an abnormality in intellectual development. or have epileptic seizures, etc.
This disease usually manifests itself from birth to adulthood. Each person will have different symptoms. Some pediatric patients may have growth retardation. In some cases, parents can notice from the unusual shape. and showing signs of illness such as enlarged stomach due to enlarged spleen, liver, blood system symptoms, anemia, low platelet count causing the wound to bruise and bleed more easily than usual Some patients may experience bone pain. have thin bones easily broken bones Information on Gosher disease can be found at For more information on rare diseases or hereditary LSD, please visit http://www.lsdthailand.com
www.thelancet.com/journals/lancet/article/PIIS0140-6736(20)30457-8/fulltext
https://eng.nhso.go.th/view/1/DescriptionNews/24-Rare-Diseases-Added-to-UCS-But-Challenges-Remain/292/EN-US
Takeda is a global biopharmaceutical company driven by research and development. Headquartered in Japan, Takeda is committed to innovating innovative treatments for a healthy and bright future for people around the world. It focuses on research and development in four disease groups: oncology, rare diseases and hematology. neuroscience and the gastrointestinal tract (GI) and also targets research and development investments in plasma- and vaccine-based products. different in people’s lives To offer new options for treatment, research and development to create more effective and diverse treatments. Our employees are committed to improving the quality of life for patients and working with local public health and healthcare partners in more than 80 countries and regions around the world.
Takeda Thailand was established in 1969 as the second branch under Takeda’s global expansion plan. Takeda Thailand’s core business unit is comprised of innovative solutions that seek life-changing treatments for patients. Under the main business groups are gastrointestinal diseases, oncology, genetic and immune rare diseases and a vaccine that is currently in progress