Acadia Pharmaceuticals said that drug regulators in Europe are leaning toward rejecting its application for a neurodevelopmental disorder treatment. The company said Monday it was informed by a ...

Williams-Beuren syndrome is a rare, congenital disease in which the main morbidity and mortality comes from obstructions, or stenoses, in specific arteries. When these obstructions involve the aorta, ...

MILAN--(BUSINESS WIRE)--With the decision by the U.S. Food and Drug Administration and the European Commission to grant market authorization for Waskyra™, a gene therapy to treat Wiskott-Aldrich ...

Encoded Therapeutics, Inc. ("Encoded"), a clinical-stage biotechnology company developing precision genetic medicines for severe neurological disorders, today announced that the U.S. Food and Drug ...

In the Neurology Department at Allegheny General Hospital’s Hemlock Building, Abigail Bailey’s body folded forward in her chair. Her muscles tightened as if someone had forced the air from her lungs.

BAY 3401016 is an investigational monoclonal antibody with potential to block a protein called Semaphorin 3A (Sema3A), which is thought to be involved in the progression of kidney damage in Alport ...

Biologics, including GLP-1 analogs and PCSK9 inhibitors, are advancing treatment options for metabolic syndrome components like type 2 diabetes and dyslipidemia. GLP-1 receptor agonists improve ...

South Korean scientists recently determined that patients with restless legs syndrome (RLS) showed approximately double the Parkinson’s disease incidence. If that doesn’t sound like good news, it’s ...

Delgocitinib has emerged as a promising treatment for CHE, addressing unmet needs and enhancing understanding of this complex condition, notes Raj Chovatiya, MD, PhD, MSCI. Chronic hand eczema (CHE) ...