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...
by Peter Ciszewski | Mar 15, 2022
Kathie Bishop, PhD, Chief Scientific Officer, Acadia Pharmaceuticals, discusses the proposed mechanism of action of trofinetide, an orphan drug in development to treat Rett syndrome. Rett syndrome is a rare progressive neurodevelopmental condition that...
