The Cystic Fibrosis Foundation and Vertex Pharmaceuticals of Cambridge, Massachusetts have been working closely together to develop and trial a new drug which treats the underlying causes of cystic fibrosis (CF), and have announced that initial trials have been very successful.
It is estimated that around 30,000 US citizens and 8,500 in the UK suffer with CF, an inherited disease which is caused by a mutated gene. The disease attacks the body’s organs with a thick mucus like substance, especially the lungs and digestive system which makes it extremely difficult for sufferers to breathe properly or digest food.
Drugs currently available only target the symptoms of the disease whereas Vertex’s new drug attempts to correct the malfunctioning protein that allows CF to flourish.
Patients involved in the study reported a vast improvement in their breathing and also began to gain weight.
A few facts about Cystic Fibrosis.
- It is one of the most life threatening hereditary diseases.
- It is estimated that 1 in 25 people are carriers for the gene that causes CF.
- If a child is conceived by two carriers it has a 1:4 chance of developing CF.
- Cystic Fibrosis affects all internal organs, but especially the digestive tract and lungs. It clogs them with a sticky mucus that makes it very difficult to breathe or digest food, starving the body of vital nutrients.
- Five babies are born every week with Cystic Fibrosis.
- Two young lives are lost every week as a result of Cystic Fibrosis.
- Only around half of Cystic Fibrosis sufferers can expect to live beyond 38 years of age but improvements in treatments and medicines mean that someone born today with the disease could live longer.
- The most famous person to suffer with CF was Albert Einstein.
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images: nlm.nih.gov, mlahanas.de