Print view
 

Studies Yield ‘Impressive’ Results in Fight Against Cystic Fibrosis

“These findings indicate that it may soon be possible to offer safe and effective molecularly targeted therapies to 90 percent of persons with cystic fibrosis,” wrote the director, Dr. Francis S. Collins, who led the team that in 1989 identified the gene that causes the genetic disease affecting the lungs and digestive system. “This should be a cause for major celebration,” he wrote in the Thursday editorial.

The two studies, published by The New England Journal of Medicine and The Lancet, describe the results of clinical trials of a three-drug therapy for people with cystic fibrosis, which causes thick mucus to build up in the lungs and other parts of the body and drastically shortens life expectancy. Both studies found that, among other things, patients who received the therapy showed improvement in a key measure of lung capacity, findings that Dr. Collins described in a separate N.I.H. blog post as “striking.”

The studies were conducted with funding from Vertex Pharmaceuticals, which developed the triple-combination drug therapy, known as Trikafta. Last week, the Food and Drug Administration approved Trikafta for use with about 90 percent of patients with cystic fibrosis — those who have the most common cystic fibrosis gene mutation and are 12 or older.

Read the full aricle here.

Autor: Niraj Chokshi   Quelle: The New York Times, 04.11.2019
"Going International promotes access to education and training for all regardless of social, geographic and national borders."
Weitere News
Johns Hopkins University_COVID-19 - Live map shows spread of the virus
New WHO courses for COVID-19 available
International Nurses Day: Nurses deserve praise, thanks, protection amid COVID-19
Will 2020 Be the Year That Medicine Was Saved?
'I feel fear and guilt': an NHS junior doctor on the effect of getting Covid-19
Coronavirus distancing may need to continue until 2022, say experts
What Covid-19 Is Teaching Us about Inequality and the Sustainability of Our Health Systems
Africa's Leaders Forced to Confront Healthcare Systems they Neglected for Years
Coronavirus Makes Inequality a Public Health Issue
APHA Opposes Trump Move to Cut Essential Who Funding
Earth Day 2020
What does love do to our brains?
COVID-19 Resource Centre of the Lancet
‘Social recession’: how isolation can affect physical and mental health
Doctors Fear Bringing Coronavirus Home: ‘I Am Sort of a Pariah in My Family
How To Support People In Health Care Working During Coronavirus
Health officials warn US government does not have enough stockpiled medical equipment to deal with coronavirus
How infants benefit from a bilingual home
Human Trafficking – It Came Disguised as the Opportunity of a Lifetime
Study finds functional medicine model is associated with improvements in health-related QOL
NHS treating 5,000 diabetics a day as one in 10 patients now suffer with illness, figures reveal
A ‘Cure’ for Ebola but Will it Stop the Outbreak if People Won’t Get Treatment?
The Role of Women’s Organisations in Crisis-Settings
CRA shortage is worsening while demand grows – a vicious circle which opens masses of job opportunities
Why Doctors Hate Their Computers
Junior hospital doctors
90–90–90 Treatment for All
Incident Management System
Incident Management System
An Economy for the 99%
European Virus Archive goes global
Establishing a Global Vaccine-Development Fund

Newsletter Sign Up

 

AMREF - African Medical and Research FoundationCharité International AcademyÄrzte der WeltOÖ Gebietskrankenkasse, Referat für Wissenschaftskooperation Hilfswerk AustriaHelix - Forschung & Beratung WienAnästhesie in Entwicklungsländern e. V.Österreichische Akademie der Ärzte