I can extract side effects.
Chapter 165 Selecting a Route
Chapter 165 Selecting a Route
Everyone may have their own understanding of what the meaning of a drug exists in this world.
Pharmaceutical companies feel that the existence of drugs is their own achievement and a cash cow, and they must squeeze the maximum profit possible.
Scientists believe that this is a treasure bestowed by nature on human beings. It belongs to all human beings. Of course, not everyone has the means to obtain it. Just like farming to gain harvest, obtaining medicine also requires a price, but it is by no means unreachable. and existence.
Patients feel that the right to life is above all things. For their own lives, they have the right to take any means to obtain medical treatment. Some patients even feel that the right to health is part of natural human rights. Can't afford it, sorry, it's not his problem, it's the society's problem.
A drug, like its molecular model, has a complex three-dimensional structure, and each side is different.
Each sees only one side of it, and thus has its own interpretation.
After all, many medicines do exist in ancient times. Most of them come from the embrace of nature, but their uses were accidentally discovered by humans hundreds of thousands of years ago. Who can monopolize such medicines?
With the development of science and technology, the development of technology is getting faster and faster, but medicine has its own uniqueness.
Its basic principles and discoveries all come from the exploration of the human body.
Its important research requires clinical trials and use on humans before it can be verified and useful.
Its final result is meaningful only when it is applied to people.
It comes from people, it is taken from people, and it is used for people.
It belongs to all mankind.
In any case, at this moment, in Sanqing's biological laboratory, when Tang Que spoke his mind.
He said the deafening words: If no one can afford the medicine, it loses its meaning of existence.
The applause was louder than ever.
Thunderous applause rang out wave after wave, and the uninterrupted strong sound waves stirred up everywhere, as if they were going to blow the high ceiling away.
Every scientific research scholar will also think deeply about the significance of his work.
Maybe they want to swim in the ocean of truth, maybe they want to be famous forever, maybe they want to be recognized by their peers and the public, maybe they want to be crowned with the crown of fame and wealth.
But, in any case, the meaning will not be purely monetary.
When a scientist's achievement cannot be used by others, it loses the meaning of existence. It can only be blown away by the wind like a fallen leaf and disappear in the dust of history.
Just like the gene therapy drug that has been on the market for four years and only sold one copy, it eventually failed to make ends meet and was withdrawn from the market sadly.
This is of course a result that no researcher wants to see.
Although, this result is capital.
If the researcher can make the decision, he may want to reduce the price of this drug to a level that patients can afford.
I also don't want to see my achievements being completely abandoned because of the price.
The pearl is covered with dust, and the treasure sinks to the bottom.
This is the loss of human beings and a waste of scientific research power.
As for China, since ancient times, there has been a tradition that the people are the sky. Every person who grows on this soil always has a feeling of compassion in his heart.
Everyone is like a dragon, which is the ideal of every Chinese.
It is also the ideal of Huaxia Medicine staff not to let a single patient go.
Now, in Sanqing, they have found the same belief and gathered together for the same ideal.
How can you not tremble in your soul and your mind is turbulent.
Everyone flushed with excitement, loud cheers accompanied by applause, mixed with some whistles.
It's like treating a serious workplace as a entertainment room for star chasing.
Tang Que clapped his hands abruptly, and the scene immediately fell silent.
"Okay, I'm not asking you to do charity, so don't be so excited. The medicine you research can't be sold for money, and the boss will definitely hack me to death."
"Hehehe." Everyone let out a burst of good-natured laughter.
"Three listed drugs, Biogen's drug has entered the medical insurance, and the price has also dropped. We don't need to attack."
"And this drug also has its limitations. First of all, it cannot cure the root cause. It mainly increases the expression level of neuronal survival protein (SMN protein) through messenger RNA (mRNA). Patients need to take medicine for life, and the way of administration is very inhumane."
"Because the antisense oligonucleotides used in this drug are not easy to pass through the blood-brain barrier, it can only be administered intrathecally. Patients have to undergo lumbar puncture injections each time, and each injection can last for three months. I think We should be more pursuing in the way of drug delivery, and consider the replacement of patients."
"So let's take a look at the treatment methods and molecular mechanisms of action of the other two sky-high drugs."
"After all, being able to sell it at such an expensive price, and insisting on refusing to lower the price, there must be something special about it."
"As for me, I like to conquer such opponents and challenge the highest difficulty."
"Next, let's analyze Novartis' 1400 million RMB high-priced drug and Roche's oral high-priced drug."
"It is well known that spinal muscular atrophy (SMA) is a rare inherited neuromuscular disorder and a leading genetic cause of infant death. Its pathogenesis is rooted in the absence or out of order."
"There are two genes that can produce SMN protein in the human body, namely the SMN1 gene and the SMN2 gene, but the SMN1 gene plays a dominant role, and the SMN2 gene only produces about 10% of a small amount of SMN protein."
"Once the SMN1 gene malfunctions, it can lead to the inability of the patient to produce enough SMN, which in turn affects the patient's movement, breathing, swallowing, and multiple organs such as the spleen, heart, pancreas, and even threatens the patient's life."
"SMA can be divided into several types based on the degree of loss of SMN protein levels. Among them, the most severe type I SMA patients usually die due to rapid neuronal death and muscle damage, and most die by 24 months. Permanent respiratory support."
"According to the pathogenic mechanism, there are two different paths for the treatment of SMA. One is to target the SMN1 gene and directly introduce the gene encoding normal SMN1 through the vector, which is the gene therapy adopted by Novartis' Z drug."
"The second is to target the SMN2 gene, and promote the normal expression of SMN2 in patients to realize the production of SMN protein. This is Roche's oral liquid E drug."
"Theoretically speaking, SMN1 gene replacement therapy is one of the most reasonable and effective methods for the treatment of spinal muscular atrophy, and its advantage is that it can obtain clinical efficacy only through a single intravenous injection."
"Novartis' Z is one such drug that delivers a copy of the SMN1 gene to muscle cells, enabling the production of SMN protein. The defective gene for SMN1 is not removed and may continue to be transcribed, with unknown downstream effects."
"This treatment method is like a vaccine, introducing the correct SMN1 gene into the host through a highly safe viral vector, thereby infecting the motor neuron cells so that the newborn cells can produce SMN protein. Only one injection is needed. The patient's body can continuously produce SMN protein, which can cure the disease in a sense."
"Of course, the second method is also very good. By regulating the splicing of SMN2 gene mRNA through small molecule binders, it can express the mRNA level of normal SMN protein, thereby alleviating the symptoms of SMA patients."
"It has to be dosed according to the patient's weight, and it needs to be taken continuously. This is an ideal drug for a pharmaceutical company. The patient is like a cash cow, and the money is continuously provided. It stands to reason that, Drug prices like this should be affordable, otherwise what's the point."
"After all, it's not like Novartis' Z drug, which is a one-shot drug that will never cause future troubles. At least it will cure the disease with one injection. It is more expensive for its own reason."
"It is such a drug that is taken for life, who gave it the courage to sell it so expensive? Don't you understand the truth of the long flow of water, so you want to use up the water?"
"I don't like this kind of arrogance the most. Next, we will take a two-pronged approach, starting with these two solutions, and come up with our own unique medicine."
Tang Que narrated these contents eloquently, explaining them clearly and easily.
Everyone understands their goals and direction of efforts.
(End of this chapter)
Everyone may have their own understanding of what the meaning of a drug exists in this world.
Pharmaceutical companies feel that the existence of drugs is their own achievement and a cash cow, and they must squeeze the maximum profit possible.
Scientists believe that this is a treasure bestowed by nature on human beings. It belongs to all human beings. Of course, not everyone has the means to obtain it. Just like farming to gain harvest, obtaining medicine also requires a price, but it is by no means unreachable. and existence.
Patients feel that the right to life is above all things. For their own lives, they have the right to take any means to obtain medical treatment. Some patients even feel that the right to health is part of natural human rights. Can't afford it, sorry, it's not his problem, it's the society's problem.
A drug, like its molecular model, has a complex three-dimensional structure, and each side is different.
Each sees only one side of it, and thus has its own interpretation.
After all, many medicines do exist in ancient times. Most of them come from the embrace of nature, but their uses were accidentally discovered by humans hundreds of thousands of years ago. Who can monopolize such medicines?
With the development of science and technology, the development of technology is getting faster and faster, but medicine has its own uniqueness.
Its basic principles and discoveries all come from the exploration of the human body.
Its important research requires clinical trials and use on humans before it can be verified and useful.
Its final result is meaningful only when it is applied to people.
It comes from people, it is taken from people, and it is used for people.
It belongs to all mankind.
In any case, at this moment, in Sanqing's biological laboratory, when Tang Que spoke his mind.
He said the deafening words: If no one can afford the medicine, it loses its meaning of existence.
The applause was louder than ever.
Thunderous applause rang out wave after wave, and the uninterrupted strong sound waves stirred up everywhere, as if they were going to blow the high ceiling away.
Every scientific research scholar will also think deeply about the significance of his work.
Maybe they want to swim in the ocean of truth, maybe they want to be famous forever, maybe they want to be recognized by their peers and the public, maybe they want to be crowned with the crown of fame and wealth.
But, in any case, the meaning will not be purely monetary.
When a scientist's achievement cannot be used by others, it loses the meaning of existence. It can only be blown away by the wind like a fallen leaf and disappear in the dust of history.
Just like the gene therapy drug that has been on the market for four years and only sold one copy, it eventually failed to make ends meet and was withdrawn from the market sadly.
This is of course a result that no researcher wants to see.
Although, this result is capital.
If the researcher can make the decision, he may want to reduce the price of this drug to a level that patients can afford.
I also don't want to see my achievements being completely abandoned because of the price.
The pearl is covered with dust, and the treasure sinks to the bottom.
This is the loss of human beings and a waste of scientific research power.
As for China, since ancient times, there has been a tradition that the people are the sky. Every person who grows on this soil always has a feeling of compassion in his heart.
Everyone is like a dragon, which is the ideal of every Chinese.
It is also the ideal of Huaxia Medicine staff not to let a single patient go.
Now, in Sanqing, they have found the same belief and gathered together for the same ideal.
How can you not tremble in your soul and your mind is turbulent.
Everyone flushed with excitement, loud cheers accompanied by applause, mixed with some whistles.
It's like treating a serious workplace as a entertainment room for star chasing.
Tang Que clapped his hands abruptly, and the scene immediately fell silent.
"Okay, I'm not asking you to do charity, so don't be so excited. The medicine you research can't be sold for money, and the boss will definitely hack me to death."
"Hehehe." Everyone let out a burst of good-natured laughter.
"Three listed drugs, Biogen's drug has entered the medical insurance, and the price has also dropped. We don't need to attack."
"And this drug also has its limitations. First of all, it cannot cure the root cause. It mainly increases the expression level of neuronal survival protein (SMN protein) through messenger RNA (mRNA). Patients need to take medicine for life, and the way of administration is very inhumane."
"Because the antisense oligonucleotides used in this drug are not easy to pass through the blood-brain barrier, it can only be administered intrathecally. Patients have to undergo lumbar puncture injections each time, and each injection can last for three months. I think We should be more pursuing in the way of drug delivery, and consider the replacement of patients."
"So let's take a look at the treatment methods and molecular mechanisms of action of the other two sky-high drugs."
"After all, being able to sell it at such an expensive price, and insisting on refusing to lower the price, there must be something special about it."
"As for me, I like to conquer such opponents and challenge the highest difficulty."
"Next, let's analyze Novartis' 1400 million RMB high-priced drug and Roche's oral high-priced drug."
"It is well known that spinal muscular atrophy (SMA) is a rare inherited neuromuscular disorder and a leading genetic cause of infant death. Its pathogenesis is rooted in the absence or out of order."
"There are two genes that can produce SMN protein in the human body, namely the SMN1 gene and the SMN2 gene, but the SMN1 gene plays a dominant role, and the SMN2 gene only produces about 10% of a small amount of SMN protein."
"Once the SMN1 gene malfunctions, it can lead to the inability of the patient to produce enough SMN, which in turn affects the patient's movement, breathing, swallowing, and multiple organs such as the spleen, heart, pancreas, and even threatens the patient's life."
"SMA can be divided into several types based on the degree of loss of SMN protein levels. Among them, the most severe type I SMA patients usually die due to rapid neuronal death and muscle damage, and most die by 24 months. Permanent respiratory support."
"According to the pathogenic mechanism, there are two different paths for the treatment of SMA. One is to target the SMN1 gene and directly introduce the gene encoding normal SMN1 through the vector, which is the gene therapy adopted by Novartis' Z drug."
"The second is to target the SMN2 gene, and promote the normal expression of SMN2 in patients to realize the production of SMN protein. This is Roche's oral liquid E drug."
"Theoretically speaking, SMN1 gene replacement therapy is one of the most reasonable and effective methods for the treatment of spinal muscular atrophy, and its advantage is that it can obtain clinical efficacy only through a single intravenous injection."
"Novartis' Z is one such drug that delivers a copy of the SMN1 gene to muscle cells, enabling the production of SMN protein. The defective gene for SMN1 is not removed and may continue to be transcribed, with unknown downstream effects."
"This treatment method is like a vaccine, introducing the correct SMN1 gene into the host through a highly safe viral vector, thereby infecting the motor neuron cells so that the newborn cells can produce SMN protein. Only one injection is needed. The patient's body can continuously produce SMN protein, which can cure the disease in a sense."
"Of course, the second method is also very good. By regulating the splicing of SMN2 gene mRNA through small molecule binders, it can express the mRNA level of normal SMN protein, thereby alleviating the symptoms of SMA patients."
"It has to be dosed according to the patient's weight, and it needs to be taken continuously. This is an ideal drug for a pharmaceutical company. The patient is like a cash cow, and the money is continuously provided. It stands to reason that, Drug prices like this should be affordable, otherwise what's the point."
"After all, it's not like Novartis' Z drug, which is a one-shot drug that will never cause future troubles. At least it will cure the disease with one injection. It is more expensive for its own reason."
"It is such a drug that is taken for life, who gave it the courage to sell it so expensive? Don't you understand the truth of the long flow of water, so you want to use up the water?"
"I don't like this kind of arrogance the most. Next, we will take a two-pronged approach, starting with these two solutions, and come up with our own unique medicine."
Tang Que narrated these contents eloquently, explaining them clearly and easily.
Everyone understands their goals and direction of efforts.
(End of this chapter)
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