by Peter Ciszewski | Sep 6, 2022
The US Food and Drug Administration (FDA) has approved spesolimab to treat adults with generalized pustular psoriasis (GPP) flares. GPP is a rare skin disorder in which neutrophils accumulate in the skin that result in painful, sterile pustules. GPP flares can lead...
by Peter Ciszewski | Sep 6, 2022
Eric LeFebvre, MD, Chief Medical Officer at Pliant Therapeutics, describes the mechanism of action of PLN-74809, an orphan drug in development for treating patients with idiopathic pulmonary fibrosis (IPF). IPF is a chronic, progressive, fibrosing lung disease...
by Peter Ciszewski | Aug 31, 2022
Deirdre Kelly, MD, FRCP, FRCPI, FRCPCH, Consultant Pediatric Hepatologist, Liver Unit, Birmingham Women’s and Children’s Hospital and University of Birmingham, discusses the diminished quality of life associated with being a caregiver for a progressive familial...
by Peter Ciszewski | Aug 19, 2022
Kala McWain, mother of a child with phenylketonuria (PKU), talks about the disease and the challenges of finding and adhering to a very expensive diet. PKU is a rare genetic metabolic disorder that results in reduced activity of phenylalanine hydroxylase that...
by Peter Ciszewski | Aug 18, 2022
The U.S. Food and Drug Administration (FDA) has approved betibeglogene autotemcel, a one-time gene therapy to treat patients with beta‑thalassemia who require regular red blood cell (RBC) transfusions. This is the first gene therapy to be approved for this condition....
