Focus on rare diseases and let the rare be seen

Mondo Health Updated on 2024-02-29

International Day of Rare Diseases.

Today is the 17th International Day of Rare Diseases. The World Health Organization defines rare diseases as the number of people who have a disease of 0 out of the total population065 to 01 Most of them are congenital, chronic, and often life-threatening.

Baby Panda" (Gaucher Disease), "Beauty Disease" (Multiple Sclerosis), "Porcelain Doll" (Osteogenesis Imperfecta) ......In addition to these rare diseases that we have heard of, several even rarer cases have been recorded in Beijing Satellite TV's "Lifeline".

A 22-year-old girl has thousands of tumors in her body.

Neurofibromatosis.

Neurofibromatosis, as the name suggests, may grow tumors wherever there are nerves on the body, if there are many bumps on the epidermis, or a lot of tumors in the spinal canal, it is called neurofibromatosis type 1, also known as plexiform cell tumor. This pervasive and painful disease makes all the places in the body that can be touched by nerves become fertile ground for tumor growth.

22-year-old Dandan suffered from neurofibromatosis, but he was half-paralyzed in bed at the age when he should be young, and hundreds of tumors made Dandan's young body feel like being bitten by ants all the time, Dandan was so painful that he could only kneel on the bed with half paralysis, and even his neck could not twist freely.

Most children do not live to be 10 years old.

Mucopolysaccharidosis.

This is a rare disease with congenital metabolic abnormalities, with an incidence of only 1 in 100,000, and the main incidence group is children, so children with this rare disease are often called "sticky babies".

6-year-old Xiao Sui Xin was diagnosed with mucopolysaccharidosis type 1, and his body stayed at the age of 3 and no longer developed.

Children with mucopolysaccharidosis are unable to secrete enzymes that degrade acidic mucopolysaccharides, resulting in the accumulation of mucopolysaccharides in the body, which eventually develops into organ failure. Most of them are short in stature, rough in appearance, corneal cloudiness, joint deformity, enlargement of the liver and spleen, heart valve disease, ear, nose and throat lesions, and often end up in wheelchairs, and most of them do not live to be 10 years old.

10 intensive care units in 2 years.

Asphyxia thoracic dysplasia syndrome.

Asphyxia thoracic dysplasia syndrome is a rare autosomal recessive, achondroplasia disorder that occurs in early infancy. According to statistics, the mortality rate of such patients can reach 60% to 80% in the first few years of life, and the mortality rate of the few patients who have the opportunity to undergo surgery** can also reach 30%.

4-year-old Xiao Huihui was diagnosed with "asphyxiated thoracic dysplasia syndrome", entered the ICU 10 times in 2 years, and spent more than 400 days in the intensive care unit.

Xiao Huihui's chest cavity is divided into three parts by the deformed and developed ribs: the anterior bulge area has been completely occupied by the enlarged heart, while the left and right ribs, which should be full, have been squeezed into a thin barrel shape by the deformed and sunken ribs and the stunted back ribs, resulting in no room for development of the lungs on both sides. As a result, Xiao Huihui suffered from respiratory failure due to pneumonia many times.

A 15-year-old boy has a heart like an 80-year-old.

Baker progressive muscular dystrophy.

15-year-old Gao Gao went to the hospital for a check-up due to sudden heart failure, originally thought it was just heat stroke or stomach cold, but was sentenced to "death" by local doctors: grade 4 heart failure, sudden death at any time. When doctors traced the condition, they discovered the underlying cause of high and high heart failure, Beck progressive muscular dystrophy.

Beck progressive muscular dystrophy occurs in about 19,000 people.

1. Rare genetic diseases in which women carry pathogenic genes but do not develop the disease, and men have 50% of the likely diseases. Once the disease occurs, there will be gradual muscle weakness, atrophy, difficulty walking, dyspnea, and some patients will have severe dilated cardiomyopathy, and there is no effective method and drug in the world.

likes basketball, and originally planned to be a sports student, but now he can only lie in bed and look at his phone quietly.

As of February 2022, there are more than 7,000 known rare diseases worldwide. There are more than 20 million patients with rare diseases in China, with more than 200,000 new patients every year.

Pay attention to rare diseases, let the rare be seen, so as to get early**. It is believed that with the continuous development of global medical technology, more rare disease patients will be able to obtain ** and ** opportunities.

Related Pages