AzurRx BioPharma is requesting a new patient arm be added to its ongoing Phase 2b trial investigating MS1819 capsules as a…
Articles by Marisa Wexler, MS
The European Commission has approved Kalydeco (ivacaftor), by Vertex Pharmaceuticals, to treat infants as young as four months…
The National Organization for Rare Disorders (NORD)’s RareLaunch training program will host two days of…
Oral lenabasum failed to significantly reduce the frequency of pulmonary exacerbations in people with cystic fibrosis (CF) treated in…
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…