New Stem Cell Method Could Advance Research Into Spinal Muscular Atrophy by Madaline Spencer| Published on: Oct 15, 2026 (+++)

New Stem Cell Method Could Advance Research Into Spinal Muscular Atrophy by Madaline Spencer| Published on: 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. https://checkrare.com/new-stem-cell-method-could-advance-research-into-spinal-muscular-atrophy/ Ultragenyx Sells PRV for $210M | The CheckRare Brief Ep 11 by CheckRare Staff| Published on: Oct 14, 2026 In this episode, we discuss Ultragenyx’s Priority Review Voucher that sold for $210 million, the efficacy of newborn screening on MLD, and ethical considerations needed to design and conduct clinical trials. https://checkrare.com/ultragenyx-sells-prv-for-210m-the-checkrare-brief-ep-11/ FDA Approves Gazyva for Idiopathic Nephrotic Syndrome, Offering a New Treatment Option by Madaline Spencer| Published on: 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). https://checkrare.com/fda-approves-gazyva-for-idiopathic-nephrotic-syndrome-offering-a-new-treatment-option/