Oral lenabasum failed to significantly reduce the frequency of pulmonary exacerbations in people with cystic fibrosis (CF) treated in…
Articles by Marisa Wexler, MS
A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like…
A Phase 2 clinical trial of oral lenabasum in people with cystic fibrosis (CF) failed to meet its primary goal of…
Oral antibiotics are just as effective as intravenous (into-the-vein) antibiotics at killing Pseudomonas aeruginosa bacteria in people with…
The U.S. Food and Drug Administration (FDA) has approved Vertex Pharmaceuticals‘ Kalydeco (ivacaftor) as a treatment…
The European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended extending the licenses…
Adding pancreatic enzymes to “pre-digest” food was found to be an effective way to ensure nutrition in an infant with…
The European Medicines Agency (EMA) has validated an application that seeks to expand the approval of Kaftrio (…
Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) is safe for use in children with cystic fibrosis (CF) as young as age…
A series of new research projects will seek to better understand how the immune system responds to transplanted organs, including…