precisionmedicineonline2 天
CHMP Recommends Vertex's Kaftrio, Kalydeco Combo for Additional Cystic Fibrosis Patients
The committee supports approving the CFTR modulator for patients 2 years and older with at least one non-class I CFTR gene mutation.
Vertex Pharma wins EU backing to expand label for Kaftrio cystic fibrosis drug
Vertex Pharmaceuticals (VRTX) stock in focus as company secures EU backing to expand label for its cystic fibrosis therapy ...
Healio2 天
Food allergy less common among children with cystic fibrosis
In a cohort of nearly 300 pediatric patients with cystic fibrosis, food allergy was found in less than 4% of the population, ...
pulmonologyadvisor2 天
Cystic Fibrosis Lung Transplant Data Suggest Racial Disparities in HEMT Benefits
Racial disparities in lung transplantation rates in CF suggest that HEMT has benefitted White patients more than Black and Hispanic patients.
Sionna Therapeutics to Present at the 45th Annual TD Cowen Health Care Conference
Sionna Therapeutics, Inc. (Nasdaq: SION), a clinical-stage biopharmaceutical company on a mission to revolutionize the ...
EurekAlert!4 天
CRISPR technologies: Revolutionizing regenerative medicine
A new review in Engineering reveals that CRISPR technologies are revolutionizing regenerative medicine. Scientists from ...
Cystic Fibrosis Foundation6 天
More than 150 Advocates Call on Congress to Protect the NIH, FDA, and Medicaid
Volunteers shared personal stories about life with CF while urging members of Congress to protect patients and progress.
Seeking Alpha2 天
VRTX Vertex Pharmaceuticals Incorporated
It markets TRIKAFTA/KAFTRIO for people with CF with at least one F508del mutation for 2 years of age ... of acute pain in adults; VX-522, a CFTR mRNA therapeutic designed to treat the underlying ...