November 03, 2019 – Cystic fibrosis (CF) is a lethal, inherited genetic disease affecting more than 70,000 people worldwide. It is usually diagnosed in childhood and causes abnormal buildup of thick mucus in the lungs and digestive tract that leads …

In Caribbeans cystic fibrosis (CF) is driven by very rare CFTR mutations Read more »

August 13, 2019 – A very significant research article by the title “Allele specific repair of splicing mutations in cystic fibrosis through AsCas12a genome editing” has just appeared in the open access Journal Nature Communications. This work provide hope to patients with …

Cystic fibrosis: CFTR-gene editing may cure this genetic disease Read more »

May 18, 2017 – Ivacaftor (Kalydeco) is a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator indicated for the treatment of cystic fibrosis (CF) in patients age 2 years and older who are carriers one of the following mutations in the CFTR gene: …

Theragenomics: Ivacaftor (Kalydeco) in the CFTR mutation-based treatment of cystic fibrosis (CF) Read more »

July 2, 2015 – The following post would constitute a perfect example how theragenomic and personalized medicine may help patients who carry genetic predispositions for sometimes rather debilitating and eventually fatal diseases. Thus, the U.S. Food and Drug Administration (FDA) today …

Theragenomic medicine: Orkambi approved by the FDA as a new treatment for cystic fibrosis Read more »