such as cystic fibrosis, hemophilia, and sickle cell disease. Although you can’t always prevent getting a potentially harmful mutation or passing one on to your children, you may be able to ...

He also never complained a day in his life — no easy feat for any child, let alone one who was born with cystic fibrosis ... working properly due to mutations (harmful changes) within that ...

Compare that with a disease like cystic fibrosis, which is caused by more than 2,000 mutations in what is known ... unintentional, and even harmful errors involving large deletions or inversions ...

While many are harmless, some are known to be involved in diseases, including cystic fibrosis ... 57% of missense mutations cause no harm, while around a third (32%) may be harmful, and the ...

In this review of RNA therapies, we paint an overview of what’s going on in a field that is addressing a number of diseases.

Despite new medication, cystic fibrosis often leads to permanent lung damage. Working with an international team, researchers ...

The Massachusetts-based biotech plans to use the funds to push its candidates into mid-stage clinical trials in a space ...

Patients aged 6 to 11 years with cystic fibrosis receiving 24-week once-daily vanzacaftor/tezacaftor/deutivacaftor generally ...

As Healio previously reported, the FDA recently approved Alyftrek for patients aged 6 years and older with cystic fibrosis with certain mutations based, in part, on data from these trials ...

In December, the FDA approved Vertex’s vanzacaftor, tezacaftor and deutivacaftor (Alyftrek) for the treatment of cystic fibrosis in patients with at least one F508del mutation or another ...

Researchers have been trying for decades to stop the cells that damage the lung in people with pulmonary fibrosis. Now a team ...