Studies carried out at the Children’s Hospital and the Garrahan Hospital have shown that the treatment is safe and effective with a positive impact on patients with this disease.
CFTR (Cystic Fibrosis Transmembrane Conductance Regulator) modulators are drugs designed to correct the defect in the CFTR protein caused by genetic mutations in patients with cystic fibrosis.
The results of the generic formulation of the triple therapy treatment were presented at the European CF Society Conference (held in Glasgow, UK) from 5 to 8 June. The real-life studies were carried out at the Ricardo Gutierrez Children’s Hospital led by the Dr. Alejandro Teperhead of the institution’s Respiratory Center (MN: 58664) and at the Garrahan Hospital, by Dr. Claudio Castañoshead of the Pediatric Pulmonology Service (MN: 78503). The objective was to evaluate the real-life impact of using the Gador triple therapy treatment formulation in patients with cystic fibrosis older than 6 years, who had responsive variants and had not previously received treatment with CFTR modulators.
CFTR (Cystic Fibrosis Transmembrane Conductance Regulator) modulators are drugs designed to correct the defect in the CFTR protein caused by genetic mutations in patients with cystic fibrosis. There are different types of modulators: enhancers, which improve the function of CFTR proteins that reach the cell surface, and correctors, which increase the amount of functional proteins in the cell. These treatments aim to restore the normal function of the chloride channel and improve the patient’s health.
The main conclusions of both studies include that patients treated with generic triple therapy had significant improvements in lung function, nutritional status and quality of life, as well as a reduction in respiratory exacerbations and decreased levels of chlorine in sweat. This treatment was well tolerated, consolidating itself as a therapeutic option for patients in Argentina.
Cystic Fibrosis in numbers
- Around 60% of cases are diagnosed before the first year of life, thanks to neonatal screening.
- Between 10% and 15% of patients are diagnosed in adulthood, generally with milder forms of the disease.
- In Argentina, CF affects one in every 7,000 births, and it is estimated that more than 3,000 people suffer from this disease.
- According to the ReNaFQ (National Registry of Cystic Fibrosis), 52% are men and 80% are under 26 years of age, which highlights the prevalence of cases in young adults.
- It is estimated that one in every 40 people in Argentina is a healthy carrier of this disease.
Source: Ambito
I am an author and journalist who has worked in the entertainment industry for over a decade. I currently work as a news editor at a major news website, and my focus is on covering the latest trends in entertainment. I also write occasional pieces for other outlets, and have authored two books about the entertainment industry.
