by Madaline Spencer | Jan 29, 2026
Raffi Tachdjian, MD, Associate Clinical Professor of Medicine & Pediatrics, Division of Allergy & Clinical Immunology, University of California Los Angeles, discusses a new formulation of Orladeyo (berotralstat) for the treatment of pediatric patients with...
by Madaline Spencer | Jan 28, 2026
The US Food and Drug Administration (FDA) has approved the expanded indication of Cerezyme (imiglucerase) for the treatment of non-neuronal symptoms in adult and pediatric patients with Gaucher disease (GD) type 3. GD refers to a group of inherited metabolic diseases...
by Madaline Spencer | Jan 27, 2026
Data from the Phase 2b CORAL trial of nalbuphine extended release (ER) for the treatment of chronic cough in patients with idiopathic pulmonary fibrosis (IPF) was recently published in the Journal of the American Medical Association (JAMA). IPF is a rare, progressive...
by Madaline Spencer | Jan 26, 2026
Guy Young, MD, Children’s Hospital Los Angeles, discusses the SWITCH clinical trial design for patients with hemophilia A. Hemophilia A is an inherited bleeding disorder in which the blood does not clot normally. People with hemophilia A will bleed more than...
by Madaline Spencer | Jan 23, 2026
Christine Gustafson, Executive Director and CEO of the TED Community Organization, discusses her personal journey with thyroid eye disease (TED) and why she started the TED Community Organization. TED is a chronic endocrine, autoimmune disease characterized...
