I can extract side effects

Kunming First People's Hospital, Pediatric Ward.

Since all intravenous drugs, such as subcutaneous injection drugs, intramuscular injection drugs, etc., need to undergo clinical trials in the hospital, the hospitalization time is usually 1-3 days.

Of course, the treatment interval is the non-medication time, and patients can rest at home.

Therefore, after Cao Yang got the news of the successful development of the gene drug, he immediately took his son Xiaowei to the designated cooperative hospital of Sanqing Pharmaceutical to officially start the clinical trial.

Seeing a needle stuck into his son Xiaowei's vein, Cao Yang's expression became tense.

After all, it was the first time to use genetic medicine. Although the animal experiments were very successful, he was still a little uneasy.

According to the plan, to deliver ZFN into the body, each injection can cover a certain range of somatic cells. Somatic cells undergo genome editing, and the diseased genes in them will be repaired.

However, since the Menkes lesion gene is expressed in many places in the body, it is unlikely that a single injection will allow the therapeutic range to cover all somatic cells in the body.

In theory, patients would also need regular injections every few months.

Although the duration is relatively long, patients do not need to be hospitalized every time, they just need to get an injection in the outpatient clinic. For most patients, this will hardly cause any burden.

When the adult stem cells in the body are cured by gene editing, the new cells it produces will be healthy cells, and the patient will be completely cured.

Half an hour later, the injection was completed, and Cao Yang felt relieved when he saw that his son was fine.

Children need to stay in the ward for a few days to observe the specific curative effect.

On the third day, the doctor looked at the physical examination report and nodded with satisfaction: "The child's physical examination is all normal, and the serum copper and ceruloplasmin in the laboratory tests have also returned to normal levels. Next, you remember to give regular injections once every three months. To see the aftermath."

Cao Yang clenched his fists tightly, his heart was filled with excitement, looking at his son who was sleeping soundly, he couldn't help crying.

All the dedication and hard work finally paid off.

Son, finally saved.

Typical patients with this disease will generally die within 3 years old, and only a small number of mild cases can survive to 20 years old, so the fate of final death cannot be changed.

In the past two years, he has been chasing desperately, racing against the god of death, and finally rushed ahead of the god of death to save his son from the line of life and death.

No longer have to worry about my son not living to be 3 years old.

After the doctor left, he quickly shared the good news in the group.

The group of patients suddenly burst into cheers.

"Great, our children are all saved."

"Xiaowei looks pretty good, it seems that he is really good."

"Thank you Brother Cao, all this is thanks to you. If you hadn't traveled around and paid so much, the children would not be where they are today."

"Thank you Brother Cao."

"I also want to thank Sanqing. If it weren't for their willingness to lend a helping hand, we don't know when we will see the advent of genetic medicine."

"Fortunately, there is such a high-tech pharmaceutical company in China that can develop high-tech products such as genetic medicine. It is all because of the strength of the country that all walks of life can rise."

"Yeah, I still remember that we used to use imported medicine when we got sick, and the domestic medicine didn't work well. Now I go to the hospital to see a doctor. Some daily medicines, the doctor recommends Sanqing to me, saying that the effect is better than imported medicine. And it's cheap."

"With the rise of domestic medicine, there are now genetic medicines. I am really more patriotic than ever before, and I am proud of being born in China."

"Haha, it must be. There is no cure for this disease in foreign countries. Only in China can there be genetic medicine. Foreigners must envy us for this."

"When will the clinic officially start, we can't wait."

"Hey, wait a minute. I received a notification from Longtu. It seems that the clinical trial has officially started. Hurry up and sign up."

"So fast, I'll go right away."

The patients clicked into the APP at the speed of light, and the function of "clinical trial application" was already available.

They filled in some relevant information, signed and uploaded it, and submitted the application soon.

There were not many people, only 60 people, so they all passed quickly.

Since the Menkes disease is too rare and there are only so few people in China, when Sanqing applied for the clinical trial, it directly put the clinical phase I and phase II together. As for the clinical phase III, it may go abroad to find some patients. participated.

It is estimated that by the time it goes on the market, there will only be a few hundred patients left in the world. With such a small market, no wonder no other pharmaceutical companies are willing to do it.

That is to say, Sanqing is willing to invest in such an unpopular disease because it needs to combine the entire rare disease and conduct unified research as a single-gene genetic disease.

Otherwise, it is estimated that only Cao Yang will fight alone and be self-reliant.

Even if he finally has results, it may be several years later.

Whether Xiaowei will still be alive at that time is still a question.

Therefore, no one understands the difficulties of genetic medicine better than the family members of these children, and no one is more grateful to Sanqing than them.

Even if it is cancer, some patients can still survive after treatment, and Alzheimer's disease, the patients are all elderly people, and the chance of death is not high.

Only patients with rare diseases are babies, most of whom have no cure, and often die early before the age of five, leaving huge trauma and grief in the lives of their parents.

From this point of view, the actions of Sanqing Pharmaceutical are the reborn parents of patients with rare diseases.

No matter how grateful the family members are, they cannot be overstated.

They almost wished to make a long-lived archway for Wei Kang, and kowtowed day and night.

Sanqing soon held a press conference, announcing the launch of the first gene therapy drug in China.

As soon as the news was released, the media were shocked, and they immediately wrote a news report with great excitement and published it on the whole network.

This is big news!

Gene therapy! Isn't this something that only exists in science fiction? It has come into reality.

No matter how serious the media is, they are all shocked at this moment, and their words are astonishing.

"Shocked! Sanqing successfully developed a gene therapy drug to overcome Menkes' rare disease!"

"Shocked! Huaxia will enter the era of gene editing!"

"Shocking! How long will it take us from science fiction to reality, from gene therapy to gene evolution?"

"Talking about the future of medical care in the era of gene editing!"

"Top ten technologies that have changed human beings, the past and present of gene editing technology!"

The media also found old news about Cao Yang a year ago, conducted an in-depth interview with him and other Menkes family members, and made a special program on gene editing drugs.

Netizens were shocked by Sanqing's SMA gene drug before, but now two months later, they were shocked again.

Seeing Sanqing's research results jumping out one after another, netizens were a little numb with excitement.

"Sanqing is amazing! Mr. Wei is too awesome."

"Although there are few patients with Menkes' disease, it is extremely painful for the patients and their families. Thanks to Sanqing, who is willing to expend energy to overcome this disease, and once again saved dozens of patients."

"What a miracle it is, what a greatness, I'm just so moved."

"Cao Yang, who made his own medicine for his son, is also touching. Sure enough, in this world, no one is afraid of difficulties, but someone with a heart. Such a father is really a role model for my generation."

"How did Sanqing's genetic drug develop so quickly? It's like a hen laying eggs. I'm already numb. I can't say anything except applauding."

"+1, there is no fluctuation in my heart, I just take it for granted."

When the news spread overseas, hundreds of Menkes patients around the world were extremely excited, as if they had grasped the last straw.

Sorry, the title is wrong, it should be the first gene editing drug. Gene therapy should be the second category.