"Your daughter lives to be 4 years old at most, take it home and take good care of it."
A newborn case in Zhejiang, Jenny, was diagnosed with SMA, spinal muscular atrophy, a not uncommon "rare neonatal disease."
Parents often feedback that their children will not be able to climb in July. In fact, the common manifestations of children with SMA are: head balance disorders caused by muscle weakness, frog legs, spinal curvature, dyspnea, developmental delay, and decreased motor ability.
Most of the time, if the disease is unfortunately diagnosed, it is equivalent to a direct death sentence. However, a recent piece of good news has brought new hope for treatment for SMA neonatal patients.
First, 13 million "rare disease drugs" landed in China, and only one injection may be cured
According to the Drug Evaluation Center of the State Food and Drug Administration of China, Zolgensma (OAV101 injection) under the name of Novartis Group of Switzerland has been approved for clinical trials in mainland China and is expected to officially land in China soon.
This Swiss medicine costs more than 13 million yuan a shot, claiming to be the most expensive medicine in history. A shot costs 10 apartments, and more than 80% of people can't afford it. The reason why it is so expensive, the key behind the following points.
1. This drug uses the world's most advanced AAV gene therapy, which is high cost and cutting-edge technology. The researchers used adeno-associated virus AAV to carry therapeutic genes into specific tissues, pair them with bases and bind to mRNA, which in turn act on spinal cord motor neurons and even treat "aging."
2. This is the world's first disposable gene therapy for children with SMA. Only 1 injection is required, and children under 2 years of age may be cured.
Therefore, even if the price is very expensive, this innovative drug can enter the Chinese clinic, which still brings confidence to many families, and even many netizens call "the most beautiful negotiation".
After all, there is precedent for the price reduction of "rare diseases". On January 4 this year, more than 700,000 yuan of "Nosinasson sodium" dropped to about 33,000 yuan under the "soul bargain" of the national medical insurance representative, and after entering the medical insurance, the actual personal cost may be only 1,000 yuan. If this Swiss drug Zolgensma can also be included in medical insurance, the cost can be controlled.
Second, rare diseases are not rare! One in every 50 people carries the SMA pathogenic gene
SMA – spinal muscular dystrophy, is a chromosomal recessive genetic disease that is not uncommon clinically, with an average of one in every 50 people carrying this disease-causing gene. Peng Jing, chief physician of the Department of Pediatrics at Xiangya Hospital of Central South University, once said that about one in every 6,000 to 10,000 people is a child with SMA, and there may be 20,000-30,000 SMA patients or more in the mainland.
"This rare disease is easily confused with other diseases, such as infant weakness, which may be misdiagnosed as a movement disorder, cerebral palsy, and usually lung problems, which are easily treated as ordinary pneumonia until death." Electromyography and genetic testing are required to confirm the diagnosis. Professor Peng Jing said. To avoid SMA altogether, genetic testing must be done before birth.
With the rapid development of biotechnology, many rare diseases that originally "can only wait for death" have gradually had the hope of treatment, which is very gratifying. In fact, the development of biotechnology has not only blessed newborns with rare diseases, but also made great breakthroughs in helping human beings "delay and reverse aging".
This is how they found a very important, magical anti-aging substance! In April 2021, the top journal "Genes & Diseases" published a research review by Academician Ge Junbo of Zhongshan Hospital affiliated to Fudan University: NAD, an anti-aging substance, has great prospects. This substance can use many pathways to slow down aging, maintain youthful vitality, and even prevent cardiovascular disease. In addition, many cutting-edge institutions in the world have found that it can protect mitochondria, protect the elderly heart, and enhance metabolism. Harvard has confirmed that it has extended the lifespan of about 70-year-old laboratory mice by 30%.
Coincidentally, in the journal Aging in 2020, the team of Shai Efrati of Tel Aviv University in Israel found that 35 elderly people were given high-pressure pure oxygen for 3 months, and it was found that their telomere length stretched by 20%, and senescent cells were cleared by 37%. This is equivalent to the state of the human body back 25 years. And there are already related technical finished products "O2 ark hyperbaric oxygen chamber" landed, singer Stine Bieber bought 2 units at a time, a studio in the home, "O2 ark hyperbaric oxygen chamber" in Shanghai beijing also opened an experience store.
We have reason to believe that in the 21st century, many of the major problems of the century that were originally out of reach and could not be solved, such as "aging", will be properly solved.
Nearly 20 SMA patients have received treatment and can be expected in the future
At present, 13 million therapeutic drugs in Switzerland have been clinically approved, and another SMA treatment drug, nocinazone sodium, has also been significantly reduced after the soul bargain in January this year.
With the joint efforts of capital and technology, the new version of the national medical insurance drug catalogue landed in 11 provinces and cities such as Beijing, Shanghai, Guangzhou, Fujian, Jiangxi, Henan, etc., and nearly 20 SMA patients were injected with sodium injection of Nocinasine, and the cost was only a few thousand yuan, which greatly reduced the economic burden of the corresponding groups.
Similarly, the above-mentioned anti-aging material finished product - "Cyler" has become popular after being purchased by Jingdong since the affordability landed.
The key substance of "Cylery" was initially speculated to 12,500 yuan at the beginning of 500mg, eating more than 1.5 million a year, originally only a vassal of Li Ka-shing, Pan Shi and other super-rich people, and now it has landed in the ordinary consumer market, and the price is no longer "condescending". In jd.com's more than 5,000 comments, the praise reached 96%, and Cylery was regarded as an "artifact of intervening in aging and aging", bringing youth hope to many ordinary people.
It was the best of times. Gene therapy, nanorobots, animal organ transplantation, aging intervention and other technologies have all gone up a step. And under the blessing of capital, it is the same way - the price is getting lower and lower, the audience is getting wider and wider... Not surprisingly, the 21st century will have a qualitative leap in human life expectancy.