by Peter Ciszewski | Jul 25, 2022
Bruce Cree, MD, PhD, MAS, Associate Professor of Clinical Neurology in the Department of Neurology at the University of California San Francisco, discusses autoimmune encephalitis and the rapid development within its treatment landscape. Autoimmune encephalitis...
by Peter Ciszewski | Jul 20, 2022
Recently, we interviewed Shephard Mpofu, MD, Chief Medical Officer at Novartis Gene Therapies about the company’s clinical study, the SPR1NT trial, testing Zolgensma (onasemnogene abeparvovec) in presymptomatic patients with spinal muscular atrophy (SMA). Can...
by Peter Ciszewski | Jul 19, 2022
Eric LeFebvre, MD, Chief Medical Officer at Pliant Therapeutics, discusses positive safety and efficacy data from the phase 2a INTEGRIS-IPF clinical trial of PLN-74809 in patients with idiopathic pulmonary fibrosis (IPF). IPF is a chronic, progressive,...
by Peter Ciszewski | Jul 18, 2022
Deirdre Kelly, MD, FRCP, FRCPI, FRCPCH, Consultant Pediatric Hepatologist, Liver Unit, Birmingham Women’s and Children’s Hospital and University of Birmingham, provides an overview of progressive familial intrahepatic cholestasis (PFIC). As Dr. Kelly explains,...
by Peter Ciszewski | Jul 13, 2022
Guillaume Canaud, MD, PhD, of Necker-Enfants Malades Hospital – AP-HP, the Paris Descartes University, explains why a biopsy is needed to diagnose PIK3CA-related overgrowth syndrome (PROS). PROS is a group of rare congenital disorders that lead to the...