2024-09-07 23:00:00
In response to Law No. 27,552 on cystic fibrosis, the National Ministry of Health started in July 2023 a triple therapy to combat the disease. Currently, Gadore is the only Argentine laboratory offering a drug to treat the disease.
Cystic fibrosis falls under the National Program for Rare Diseases (EPF) within the framework of Law No. 27,552, which establishes a legal system of protection, health care, work, education, rehabilitation, social security and prevention for people suffering from this disease. People can have a better quality of life.
The pharmaceutical company has been producing drugs to treat the disease for more than seven years and now provides the treatment to 640 patients, including adults and children.
What does this disease look like?
Table of Contents
Table of Contents
“CF is a hereditary disease in which both parents must be carriers of the mutated gene. In Argentina, 1 in 7,000 births has the disease, and it is estimated that more than 3,000 people in our country have the disease. , some of whom have not yet been diagnosed, explains Dr. Maria Ferrer Respiratory Hospital.
New technology corrects cystic fibrosis in cultured human stem cells
Experts explain that this pathology is progressive in nature, affecting multiple organs (pancreas, lungs, sinuses, liver, intestines, skin, reproductive system, etc.), producing infectious complications, impairment of respiratory function, absorption Poor nutrition, malnutrition, dehydration.
This disease cannot be prevented, but it can be detected early through newborn screening or heel testing.
treat
In the framework of World Cystic Fibrosis Day, which is commemorated this Friday, Gador issued a statement in which he underlined his commitment to healthcare, “providing the community with accessible cutting-edge therapies to treat cystic fibrosis.” ”.
The entity noted that the research, evaluation and training process for the development of the molecule began in 2010, with the first phase being supported by the National Institute of Industrial Technology (INTI). He then invested in technology and equipment and went on to develop the production line as it is known today.
In 2021, it launched a treatment consisting of three CFTR modulators (triple therapy) for patients aged 6 years and older and weighing 30 kg or more. They then continue working to develop more accessible and advanced treatments.
“Thanks to new treatments, we have gone from survival rates of patients who never progressed past adolescence or young adulthood to patients who enter adulthood with a quality of life that is very close to that of the general population,” Rizzo emphasized.
Pancreatic Cystic Fibrosis or Mucoviscosity Disease Act: Provisions of New Regulations
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In late 2022, the lab expanded the product’s indications to include patients ages 6 and older without weight restrictions, allowing it to increase the number of people treated.
“In the follow-up of more than 50 children who received this therapy, we found a significant improvement in lung function and a significant reduction in the chlorine content in sweat, almost reaching very close to normal values,” explains Alejandro Teper, R. Gutiérrez Children Director of the Respiratory Center of the Hospital A. Álvarez.
He also noted that observations showed a significant increase in nutrition and a decrease in respiratory exacerbations, which means fewer hospitalizations. All of these results are evident soon after starting treatment.
“Triple therapy for the treatment of CF should be considered a ‘scientific miracle’ that has changed the lives of the vast majority of patients with this disease. The evidence is that many children on the lung transplant list are able to be taken off the list, ” Tupper concluded.
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Cystic fibrosis treatment
Cystic Fibrosis: Understanding the Disease and Its Treatment
Cystic fibrosis (CF) is a genetic disease that affects multiple organs in the body, including the lungs, pancreas, sinuses, liver, intestines, skin, and reproductive system. It is characterized by the production of thick, sticky mucus that can cause respiratory and digestive problems [[2]]. In Argentina, 1 in 7,000 births has the disease, and it is estimated that more than 3,000 people in the country have CF, with some still undiagnosed [[3]].
What is Cystic Fibrosis?
CF is a hereditary disease that requires both parents to be carriers of the mutated gene. It is a progressive disease that cannot be prevented, but it can be detected early through newborn screening or heel testing [[3]]. The disease affects multiple organs, leading to infectious complications, impairment of respiratory function, poor nutrition, malnutrition, and dehydration.
Treatment Options
The National Ministry of Health has started a triple therapy to combat CF, and currently, Gadore is the only Argentine laboratory offering a drug to treat the disease [[3]]. The pharmaceutical company has been producing drugs to treat CF for over seven years and now provides treatment to 640 patients, including adults and children.
In 2021, the lab launched a treatment consisting of three CFTR modulators (triple therapy) for patients aged 6 years and older and weighing 30 kg or more. They continue to work on developing more accessible and advanced treatments. The treatment has shown significant improvement in lung function and a significant reduction in the chlorine content in sweat, almost reaching normal values [[3]].
Living with Cystic Fibrosis
Thanks to new treatments, patients with CF can now enter adulthood with a quality of life close to that of the general population [[3]]. The disease falls under the National Program for Rare Diseases (EPF) within the framework of Law No. 27,552, which establishes a legal system of protection, healthcare, work, education, rehabilitation, social security, and prevention for people suffering from CF [[3]]. People with CF can have a better quality of life due to the accessible cutting-edge therapies provided by healthcare companies like Gadore.
Management and Care
In addition to medication, people with CF require airway clearance techniques, nutritional advice, and lung transplants in some cases [[1]]. Regular exercise and physical activity are also essential to manage the disease. The National Institute of Industrial Technology (INTI) has supported the research, evaluation, and training process for the development of the molecule since 2010 [[3]].
Conclusion
Cystic fibrosis is a complex and progressive disease that requires early detection, accurate diagnosis, and appropriate treatment. With the advancement of medical technology and the commitment of healthcare companies, people with CF can now live a better quality of life. It is essential to continue supporting research and development to find more accessible and advanced treatments for this disease.
References:
Cystic fibrosis treatment
Cystic Fibrosis: Understanding the Disease and its Treatment
Cystic fibrosis (CF) is a hereditary disease that affects multiple organs, including the pancreas, lungs, sinuses, liver, intestines, skin, reproductive system, and more. In Argentina, 1 in 7,000 births have the disease, and it is estimated that more than 3,000 people in the country have CF, with some cases still undiagnosed [[3]].
What does this disease look like?
Cystic fibrosis is a progressive disease that produces infectious complications, impairs respiratory function, and affects nutrition, leading to malnutrition and dehydration [[1]]. It cannot be prevented, but it can be detected early through newborn screening or heel testing.
New technology corrects cystic fibrosis in cultured human stem cells
Researchers have made significant progress in correcting cystic fibrosis in cultured human stem cells using new technology [[4]]. This breakthrough could lead to more effective treatments for the disease.
Treatment
The National Ministry of Health has launched a triple therapy to combat cystic fibrosis, with Gadore being the only Argentine laboratory offering a drug to treat the disease [[2]]. The pharmaceutical company has been producing drugs to treat CF for over seven years and currently provides treatment to 640 patients, including adults and children.
Pancreatic Cystic Fibrosis or Mucoviscosity Disease Act: Provisions of New Regulations
The Pancreatic Cystic Fibrosis or Mucoviscosity Disease Act aims to provide a legal system of protection, healthcare, work, education, rehabilitation, social security, and prevention for people suffering from CF [[4]].
Cystic Fibrosis Treatment
CF treatment involves chest physical therapy to help loosen and clear lung secretions, exercise to loosen mucus, stimulate coughing, and improve overall health [[1]]. Airway clearance techniques, nutritional advice, and lung transplants are also part of the treatment plan [[2]]. Close monitoring and early, aggressive intervention are recommended to slow the progression of CF, leading to a longer life [[3]].
cystic fibrosis is a complex disease that requires a comprehensive approach to treatment and management. With advancements in technology and research, there is hope for more effective treatments and improved quality of life for patients with CF.
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