by Kimberly Waller | Aug 22, 2026
The US Food and Drug Administration (FDA) has approved Pasatru (garetosmab-grts) to reduce formation of new heterotopic ossification (HO) lesions and clinician-assessed flare-ups in adults with fibrodysplasia ossificans progressiva (FOP). FOP is a disorder in which...
by Kimberly Waller | Aug 11, 2026
Caterina Garone, PhD, MD, Associate Professor at the University of Bologna, discusses safety and efficacy data on Kygevvi (doxecitine and doxribtimine) in patients with thymidine kinase 2 deficiency (TK2d). TK2d is a rare genetic disease characterized by...
by Kimberly Waller | Aug 4, 2026
Doug Kerr, MD, PhD, MBA, Chief Medical Officer of Dyne Therapeutics, discusses the initiation of the phase 3 FORZETTO trial of zeleciment rostudirsen (z-rostudirsen, also known as DYNE-251), in individuals with Duchenne muscular dystrophy (DMD) amenable to exon 51...
by Kimberly Waller | Jun 29, 2026
Vlado Perkovic, MD, Professor of Medicine and Provost at the University of New South Wales Sydney, discusses results from the VISIONARY clinical trial testing Voyxact (sibeprenlimab) in patients with IgA nephropathy (IgAN). IgAN is due to the accumulation of...
by Kimberly Waller | Jun 12, 2026
Kerrie-Anne Ho, PhD, Patient Preference Lead at UCB, discusses preferences and perceptions of acute seizure medications. Data from interviews with people with seizures (PwS) and caregiver interviews were presented at the American Academy of Neurology (AAN) 2026...
