Three-Drug Therapy for Most Common Genetic Cause of Cystic Fibrosis Found Safe and Effective in 6-11-Year-Olds

An international, open-label Phase 3 study, co-led by Susanna McColley, MD, from Ann & Robert H. Lurie Children’s Hospital of Chicago, found that a regimen of three drugs (elexacaftor/tezacaftor/ivacaftor) that targets the genetic cause of cystic fibrosis was safe and effective in 6-11-year-olds with at least one copy of F508del mutation in the CFTR gene, which is estimated to represent almost 90 percent of the cystic fibrosis population in the United States.

Genetic Mutation Could Worsen Heart Function in Duchenne Muscular Dystrophy Patients

DALLAS – Nov. 4, 2020 – A mutation in the gene that causes cystic fibrosis may accelerate heart function decline in those with Duchenne muscular dystrophy (DMD), a new study by UT Southwestern researchers suggests. The findings, published online recently in the Journal of the American Heart Association, could help doctors develop new strategies to preserve heart function in this population, potentially extending patients’ lives.

CLEVELAND CLINIC UNVEILS TOP 10 MEDICAL INNOVATIONS FOR 2021

An up-and-coming gene therapy for blood disorders. A new class of medications for cystic fibrosis. Increased access to telemedicine. These are some of the innovations that will enhance healing and change healthcare in the coming year, according to a distinguished panel of clinicians and researchers from Cleveland Clinic.

In conjunction with the 2020 Medical Innovation Summit, Cleveland Clinic announced the Top 10 Medical Innovations for 2021.

Henry Ford Health System Receives $25 Million Gift, Largest Single Donation in its History

Nationally-known developer Chris Jeffries and his wife Lisa have donated $25 million to Henry Ford Health System, the largest single gift from an individual in the health system’s 105-year history. This historic gift will rapidly accelerate the growth and expansion of Henry Ford’s Precision Medicine program, with the ultimate goal of creating a Precision Health Center. The efforts will have a robust focus on the advancement of cancer research and treatment, while also expanding to other medical specialties treating behavioral health, cardiovascular and metabolic diseases.

Modifier Gene May Explain Why Some with Cystic Fibrosis are Less Prone to Infection

People with cystic fibrosis who carry genetic variants that lower RNF5 gene expression have more mutant CFTR protein on cell surfaces. Even if the CFTR protein isn’t fully functional, it’s better than none, and may explain why some with cystic fibrosis are less prone to infection than others.