Tag Archives: rare

Albireo’s rare liver disease drug Bylvay scores back-to-back approvals in U.S., Europe

With a pair of approvals on both sides of the Atlantic, liver disease-focused biotech Albireo is off and running with its new medicine Bylvay. The oral drug, the first treatment for pruritus in patients with progressive familial intrahepatic cholestasis (PFIC), scored an FDA nod Tuesday and a European approval Monday. With the FDA’s blessing, Albireo is kicking off an “immediate”… Read More »

Vaping Leaves E-Cigarette User With Rare Lung Scarring: Study

Vape. (Photo Credits: Pixabay) New York, December 5: An e-cigarette user has been diagnosed with a rare form of lung scarring typically found in metal workers, says a new study. Doctors diagnosed the patient with hard-metal pneumoconiosis, a rare form of lung disease that causes irreparable damage, persistent coughing and breathing issues. It is typically… Read More »

New gene therapy to treat rare inherited blood condition

EMA has recommended granting a marketing authorisation in the European Union for a genetically modified product for beta-thalassaemia, a rare inherited blood condition that causes severe anaemia. Zynteglo is intended for adult and adolescent patients 12 years and older who need regular blood transfusions to manage their disease and have no matching donor for a stem cell… Read More »

Research may predict outcomes for newborns with rare disease

Up until now, scientists have understood that there are two main subtypes of the disease- known as diffuse and focal. Diffuse CHI affects the entire pancreas, whilst focal CHI affects just one area of the organ, forming a focal lesion. The team from Manchester now understand that focal CHI can be further categorised into two types- spreading focal lesions and isolated… Read More »