I can extract side effects

Haishi, Department of Neurology, Huashan Hospital.

"Duk duk duk!" Zheng Jiamin gathered his mind and knocked on the door with his daughter.

"It's you, have you finished taking the sweet medicine?" The doctor inside knew her and greeted her immediately with a smile.

"Well, hello, Dr. Liu," Zheng Jiamin nodded, and handed over the case in hand with a smile on his face.

"You are on duty today. This is a child's case. Please prescribe another course of medicine."

While speaking, she sat on a chair and held her daughter on her lap.

This is a girl about 6 years old, with two braids, looking around curiously.

While looking at the examination report, Dr. Liu asked, "Has the child's condition improved recently? How about the frequency of attacks? Does he still have a fever?"

Zheng Jiamin recalled the child's situation, and said slowly: "It's okay, the condition has been relieved in the past few years, except that you need to pay special attention when you have a fever, other times are quite normal."

"Currently, it happens once or twice a month. Generally speaking, it's not very serious. It can be relieved with a single injection of diazepam. Compared with the first attack four years ago, it's much better."

Dr. Liu nodded: "This baby's intractable epilepsy is indeed very troublesome. You must not relax in the slightest. If the seizure is severe, you must send it to the ICU for emergency treatment."

"Yeah, I know, I usually pay attention not to feed the child, nor to make her too excited, to sleep well, not to be constipated. Don't give her chocolate, coffee, or anything else, or she will die gone."

Zheng Jiamin replied earnestly: "Besides this, I usually look up information on the Internet when I have free time. I remember what Dravet syndrome is. It is said that there is no specific medicine at present. I can only take some medicines to relieve symptoms and slow down the frequency of attacks. , cannot be cured."

"After a long time, resistance to these medicines will appear, so I am careful when giving her medicines. I don't seek merit but no faults. I don't pursue no seizures at all. The frequency of seizures once or twice a month is acceptable. "

Speaking of this, she suddenly remembered something, and asked expectantly: "The midazolam nasal spray I asked you to inquire about last time, has it been introduced into China now?"

Dr. Liu adjusted his glasses, and pondered: "Midazolam, I remember it was launched abroad 7 years ago, and it is the first drug on the market that can stop epilepsy in the form of a nasal spray. There was news that it was going to enter the country, and it made a lot of noise for a while, but recently it is said that it has given up."

"Ah? Why?" Zheng Jiamin asked eagerly.

Dr. Liu explained: "The reason is this. This drug has been approved as an orphan drug abroad, but its indication is status epilepticus or multiple consecutive seizures, not Dravet syndrome, so it does not meet the 121 types in China. rare disease."

"You also know that epilepsy is a common nervous system disease, not a rare disease, so this drug cannot be imported into China according to the standards of rare disease treatment drugs, and it cannot enjoy the country's rapid introduction, exemption from clinical marketing, etc. Preferential policies."

"If this drug is to be introduced into the country as a drug for epilepsy, clinical trials will need to be done again, and the investment will surge. The company can't afford it, so it gave up."

"How could this be?" Zheng Jiamin's face was filled with sadness and disappointment.

She was looking forward to this drug at first, thinking that the introduction of it into the country was a certainty, and thought that after it was launched, it would at least be a symptomatic drug, which could greatly relieve her daughter's condition.

Never expected that it would be such a result.

However, she has no complaints about pharmaceutical companies, and can understand their desire to make money.

Now that a new drug is developed, it must be registered according to the disease with the most patients, so that more drugs can be sold. It is impossible to register according to a rare disease such as an orphan drug.

But in this way, there is no way to enjoy various convenient policies for rare diseases and save the cost of clinical trials.

It can only be said that there are gains and losses, and each has its own difficulties.

"Hey, it would be great if there are medicines that can treat the symptoms in China, so you don't have to search for imported medicines so hard."

Such thoughts emerged in her mind, and she couldn't help feeling frustrated: "Rare diseases are still not given enough attention. Only Sanqing is making generic drugs in this area in China, but Sanqing's family can't do it."

"I'll prescribe you two more boxes of clobazam." Dr. Liu said.

Zheng Jiamin came back to his senses and nodded.

Dr. Liu thought about it and asked: "Is it still Sanqing's medicine? Recently, several domestic manufacturers are making generic clobazam. The medicine is effective, the price is cheaper, and the price/performance ratio is very high..."

Zheng Jiamin interrupted him, and said in a firm tone: "It's okay, just prescribe Sanqing's clobazam. Its medicine is effective, and the child is used to it. I don't worry about other medicines."

Dr. Liu smiled, said nothing more, and finished prescribing the medicine.

Zheng Jiamin took the medicine with her daughter and took a taxi home.

Sitting in the car, looking at the familiar street scene outside the window, she suddenly felt sad, her nose was sore, and two lines of hot tears flowed from the corners of her eyes.

It has been five years since her daughter had her first epileptic seizure. She clearly remembers that it was a Saturday morning when her husband was holding the child, and the sun came in from the window, making the room very warm.

The child may be too hot, so he raised his hands uncontrollably, his eyes became dull, and his eyes began to tilt to one side, and she didn't respond to shouting.

This seizure lasted 15 minutes, and they were at a loss at first, not knowing what to do.

Seeing that her daughter's pink lips slowly began to turn purple and gray again, and there was no relief, she panicked and took a taxi to the hospital and was admitted to the ICU.

A week after she was discharged from the hospital, her daughter was sent to the hospital again because of febrile convulsions. This time she was diagnosed with Dravet syndrome, a rare childhood disease caused by a mutation in the SCN1A gene.

Their husband and wife can settle in a first-tier city like Haishi, and they are considered intellectuals, but they are also dumbfounded by this result.

Dravet syndrome? Never heard of it.

But children with refractory epilepsy caused by genetic mutations, they understand.

Zheng Jiamin is still deeply impressed. That day, when she was about to leave work, she received a call from the genetic company to formally confirm her daughter's illness.

Because she was afraid that the elderly would be worried, she dared not tell her family. She and her husband found an excuse to go downstairs for a walk, and walked around the community again and again, unable to accept the truth that her daughter was terminally ill.

After walking all night, on the way back, I passed a small garden and saw an iron gate locked from the inside.

Standing in front of the iron gate, the two felt that the whole world was completely locked by the iron gate like this garden, and their mood collapsed in an instant. They couldn't help hugging each other and crying loudly.

Later, it took half a year for the two of them to calmly accept the reality, and began to check a large number of related documents, only to find that it was actually not that scary.

Children with Dravet syndrome are often in and out of the ICU as babies, and may face paralysis and sudden death at any time.

After 2 years of age, it will be relieved a lot, and the risk will be greatly reduced.

But the epileptic seizures are uncontrollable, and she and her husband don't know when the next seizure will be and how long it will last. It may be a few minutes, it may be thirty or forty minutes, or even an hour. tremble with fear.

Although my daughter is still a frequent visitor to the ICU, the two have been able to face it calmly and are less anxious.

The only thing that worries them is that there is no specific medicine, and they can only take conventional antiepileptic drugs. Not only can they not cure the disease, but even the seizures cannot be controlled.

To some extent, it is also a terminal illness.

So, apart from keeping an eye on other medicines and making plans for the future, she didn't know what to do and could only resign herself to fate.

In the past five years, for her small family, life has been like a piece of waste paper, wrinkled and wrinkled, but there is no way to talk about it.

Epilepsy is like a big vortex, engulfing it, making that small door in her world tightly closed, so that no light can pass through.

Huashan Hospital was far away from home, and she had a lot of thoughts in her mind, and she felt a wave of tiredness unknowingly, and gradually fell into a deep sleep.

Suddenly, a buzzing vibration woke her up.

At first glance, it turned out to be Dr. Liu's call. When he was hospitalized, he wrote down his phone number and asked about his illness.

"Hi, Doctor Liu, hello, what's the matter?" Zheng Jiamin asked in a vague tone, pulling himself together.

"Tiantian, right? I have good news for you. There is a new gene drug for Dravet syndrome." Dr. Liu's voice was very excited on the phone.

"What?" Zheng Jiamin couldn't believe her ears.

Dr. Liu was also very excited and explained incessantly.

"Hehe, I just found out that Sanqing Group has newly developed an anti-epileptic gene drug. It has just started clinical trials in our hospital, but some patients in other hospitals have already received the treatment, and the feedback is that the curative effect is quite good."

"Children who received this gene therapy experienced an average reduction of about 80% in spontaneous seizures, some of whom were completely seizure-free, and the therapy did not have any negative effects on the children's cognitive abilities. Influence."

"Because this kind of drug works by reducing the excitability of abnormal neuron cells so as to return to normal, rather than targeting specific diseases, it is quite universal for epilepsy diseases, and it can also solve the patient's resistance. Drug trouble."

"I have seen the clinical phase I and phase II trial reports, and the data show that the seizures of all patients have been relieved, and the degree of relief is amazing."

"Although this gene therapy cannot completely solve the problem of SCN1A gene mutation, it should be the only treatment option for Dravet syndrome."

"If your Tiantian wants to participate, please come to the hospital to sign up."

"Great, I'll come right away." Zheng Jiamin's voice trembled with excitement.

The gene medicine of Sanqing Group is very famous, and it can be said to be like a thunderbolt. Her daughter was originally a disease caused by a gene mutation. It is very promising to carry out gene therapy from this direction.

She hung up the phone, wiped away the faint tears from the corners of her eyes, and eagerly patted the back of the front seat.

"Master, I want to change my address, please drive back for me."

"Yes, it's the place to get on the bus, Huashan Hospital."

The taxi driver also heard the conversation and knew that the treatment was important, so without saying a word, he made a U-turn at the intersection ahead and sped on the way back and forth.

Zheng Jiamin sat in the back, hugged her confused daughter tightly in her arms, and looked out the window with a smile.

This time, thinking of her daughter's illness, she was not depressed or disappointed, but full of hope.

The door in my heart seemed to be no longer closed, but opened a big gap.

The sun is pouring in, the spring is infinite, and the hope is infinite.

*****

Kunming, Sanqing Headquarters.

Wei Kang leaned back, stretched his limbs, and let out a long breath.

After reading the project information all afternoon, he was a little dizzy, but overall, the progress of each project was gratifying.

"Huh, there are a lot of drugs that have entered the clinic this year. In addition to the conventional drugs such as hypoglycemic drugs, anti-inflammatory drugs, and asthma drugs, a trump card drug has been released in the field of urology. The clinical feedback is very optimistic."

"There are also several psychiatric drugs developed last year, all of which have entered the third phase of clinical trials. The clinical cycle of psychiatric drugs is long. It is already quite good to have this progress in more than a year."

"The gene laboratory has released another king bomb, the anti-epilepsy gene drug, the effect is amazing, enough to fill many gaps in this field, and the market prospect is broad."

"Epilepsy is a disease that mainly affects children and elderly patients. There are millions of people in the country. If it can be successfully launched, it will be the greatest boon for the majority of patients."

"In addition, there are two new improved antidepressant and anti-anxiety drugs. The crystal form and molecular formula of common first-line drugs have been improved, and some new adjuvants have been added."

"Compared with similar drugs, the efficacy is second. The improvement is not too big. The main reason is that the adverse reactions are greatly reduced. According to the clinical trials, the response of patients is not bad."

"At present, among all the new drugs, these three are the most eye-catching ones. They are drugs for the treatment of chronic renal failure, genetic drugs for the treatment of epilepsy, and drugs for the treatment of social phobia."

"All of them are innovative drugs in this field. At present, no similar new drugs have been officially launched. We can step up efforts to seize the market as soon as possible."

"Other manufacturers have related research and development projects, but the effect is not as good as that of Sanqing, and it is still in the clinical stage. As long as we grab the first launch, we can form a first-mover advantage."

"Hehe, Sanqing has once again won the first place in the field of psychotropic drugs and is at the forefront. It is really gratifying."

Thinking about the clinical trial report of the new drug project, Wei Kang couldn't help but smile with relief.

Immediately, he looked at the last new drug. For this new drug, he had some ideas about the development policy, but he was still unable to make up his mind.

"The rest of the antioxidant drug is equally promising."

"Antioxidation is a very hot concept. It is associated with anti-aging. It can be described as hot cooking oil and is widely sought after."

"Anything, whether it's drugs, cosmetics, or food, as long as it's related to anti-aging, there's nothing that doesn't make money."

"People's desire to delay aging has not waned for thousands of years."

"This is a rare field where you can take everything up and down."

"It's just that for anti-aging, the clinical trial cycle will be longer."

"Generally speaking, anti-aging drugs are the most suitable for making health care products.

"However, drugs are three-point poisonous. Any drug is a double-edged sword. It is difficult to grasp the degree of this. It is best to become a prescription drug and let the doctor check it."

"Otherwise, if an old man buys it and eats it indiscriminately, and something goes wrong, the company's reputation will be lost."