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Trikafta, revolutionary cystic fibrosis drug, wins $3 Million Breakthrough Prize

Village News Staff

Researchers behind the groundbreaking cystic fibrosis treatment, Trikafta, have been honored with the prestigious $3-million Breakthrough Prize. Sabine Hadida, Paul Negulescu, and Fredrick Van Goor, of Vertex Pharmaceuticals in San Diego, developed the drug which they report has significantly improved the lives of those suffering from the genetic disorder.

Trikafta, targeting a specific protein associated with cystic fibrosis, has successfully extended the life expectancy of those affected, potentially from around 30 years to over 80 years. This innovation is being hailed as one of the most significant advancements in biomedical research in the last three decades.

Francis Collins, a geneticist and physician at the US National Human Genome Research Institute, commented on the drug's impact: "It's transformed lives. Many with cystic fibrosis, once grappling with survival, now ponder retirement."

The disease, affecting around 100,000 individuals globally, results from a gene mutation causing thick secretions, particularly in the lungs. The historic approach to treatment focused on gene modification with limited success. Trikafta's approach, however, leverages a three-drug combo that corrects the malfunctioning proteins at the disease's root. Hadida recalls the skepticism they faced but emphasized the unwavering support from the patient community.

The Breakthrough Prize ceremony, held on September 14, also recognized innovations in Parkinson's disease research, cancer immunotherapy, and advancements in physics and mathematics. Among the notable awardees were Ellen Sidransky and Andrew Singleton for their genetic discoveries related to Parkinson's disease, and John Cardy and Alexander Zamolodchikov for their work on 'conformal field theories' in physics.

Established in 2012, the Breakthrough prizes are backed by various internet moguls, including Russian-Israeli billionaire Yuri Milner and Meta's CEO, Mark Zuckerberg.

 

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