Claire’s Place Foundation has announced the launch of its fundraising campaign, called #CPFoneinamillion, to support families with…
Articles by Marisa Wexler, MS
People with cystic fibrosis (CF) who are treated with Orkambi and Symkevi (Symdeko) in real-world settings tend…
First Wave BioPharma has asked the U.S. Food and Drug Administration (FDA) for permission to start a Phase 2…
A novel gene-editing tool that allows scientists to “drag-and-drop” sequences of DNA into the genome, without inducing large cuts, could…
Vertex Pharmaceuticals recently filed an application with the U.S. Food and Drug Administration (FDA) asking that Trikafta, its…
A new test that measures the excretion of bicarbonate in urine may be used to assess the effectiveness of treatments…
Differences in levels of proteins related to wound healing, cell migration and neurological development may influence whether or…
Treatment with the triple-combination therapy Trikafta (elexacaftor/tezacaftor/ivacaftor) reduces inflammation and promotes lung tissue repair in cystic fibrosis (CF),…
People with cystic fibrosis (CF) tend to report improvements in health-related quality of life associated with treatment with Trikafta. Reports of worse mental health…
Later this month, Laurent Pharmaceuticals will meet with the U.S. Food and Drug Administration (FDA) to discuss next…