by Peter Ciszewski | Mar 24, 2022
Edward Neilan, MD, PhD, Chief Scientific and Medical Officer at the National Organization for Rare Disorders (NORD), discusses the organization’s recently established Rare Disease Centers of Excellence. As Dr. Neilan explains, there were two main goals when...
by Peter Ciszewski | Mar 23, 2022
James Howard Jr., MD, Distinguished Professor of Neuromuscular Disease and Professor of Neurology and Medicine at UNC School of Medicine, reviews the treatment landscape for myasthenia gravis. Myasthenia gravis is a chronic autoimmune neuromuscular disease...
by Peter Ciszewski | Mar 22, 2022
Stefan M. Schieke, MD, Assistant Professor of Dermatology at University of Wisconsin-Madison, discusses the role of the dermatologist in diagnosing and treating cutaneous T-cell lymphomas (CTCL). Cutaneous T-cell lymphoma belongs to the non-Hodgkin lymphoma...
by Peter Ciszewski | Mar 18, 2022
Richard Poulin, father of a young girl with aromatic L-amino acid decarboxylase (AADC) deficiency, discusses his daughter’s positive experience with an investigational gene therapy. AADC deficiency is a rare genetic disorder that affects the nervous and...
by Peter Ciszewski | Mar 16, 2022
Patrick Horn, MD, PhD, Chief Medical Officer at HemoShear Therapeutics, discusses the phase 2 HERO trial evaluating HST5040 in patients with methylmalonic acidemia (MMA) and propionic acidemia (PA). MMA and PA are rare inherited metabolic disorders in which...
