Nanobody repairs misfolded CFTR inside cells, boosting function in cystic fibrosis

A tiny antibody component could fundamentally transform the treatment of cystic fibrosis: For the first time, researchers ...
EurekAlert!

A fundamentally new therapeutic approach to cystic fibrosis: Nanobody repairs cellular defect

A tiny antibody component could fundamentally transform the treatment of cystic fibrosis: For the first time, researchers ...
AFP

Vertex Announces US FDA Approval for Label Extensions of ALYFTREK® and TRIKAFTA®, Expanding Availability of These Medicines to ~95% of All People With CF in the United States

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the U.S. Food and Drug Administration (FDA) has approved expanded use of ALYFTREK ® (vanzacaftor ...
pharmaphorum

Is pharma’s interest in orphan drugs waning?

For many years, orphan drugs for rare diseases have been a draw for pharma companies, offering high prices, swift regulatory reviews, and often extended marketing exclusivity – but there are signs ...