by Madaline Spencer | Oct 15, 2026
Kyle Loh, PhD, Associate Professor at Stanford Medicine, discusses research that could help scientists better understand spinal muscular atrophy (SMA) by generating new motor neurons from human hindbrain cells in the laboratory. SMA is a genetic...
by Madaline Spencer | Oct 14, 2026
Keisha L. Gibson, MD, MPH, pediatric nephrologist at the UNC School of Medicine, discusses the approval of Gazyva (obinutuzumab) for patients with childhood-onset idiopathic nephrotic syndrome (INS). INS is a rare, autoimmune kidney disease that is often...
by Madaline Spencer | Oct 13, 2026
The US Food and Drug Administration (FDA) has approved pirtobrutinib for adults with previously untreated chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) with no known 17p deletion. CLL/SLL are rare cancers that affect B lymphocytes. The...
by Madaline Spencer | Oct 9, 2026
Michael Benatar, MD, PhD, Department of Neurology and ALS Center, University of Miami Miller School of Medicine, discusses the use of plasma proteomics in predicting clinical manifestations of amyotrophic lateral sclerosis (ALS). ALS is a rare motor neuron...
by Madaline Spencer | Oct 8, 2026
Lukasz Jarzyna, Vice President of Rare Disease at LEO Pharma, discusses the Priority Review for dersimelagon in the treatment of erythropoietic protoporphyria (EPP) and X-linked protoporphyria (XLP). EPP and XLP are rare congenital metabolic disorders...
