by Madaline Spencer | Apr 14, 2025
Paul Bolno, MD, President and Chief Executive Officer of Wave Life Sciences, discusses positive results from the FORWARD-53 clinical trial evaluating WVE-N531 in patients with Duchenne muscular dystrophy (DMD) amenable to exon 53 skipping. DMD affects...
by Madaline Spencer | Apr 10, 2025
A new study by Clarke and colleagues published in the Journal of Clinical Endocrinology & Metabolism highlights the safety and efficacy of TransCOn PTH (palopegteriparatide) to treat adults with hypoparathyroidism. Hypoparathyroidism Hypoparathyroidism is a rare...
by Madaline Spencer | Apr 9, 2025
Richard J. Nowak, MD, Director of the Myasthenia Gravis Clinic at Yale University, discusses positive data from the MINT clinical trial evaluating Uplizna (inebilizumab-cdon) in adults with generalized myasthenia gravis (gMG). MG is a chronic autoimmune...
by Madaline Spencer | Apr 9, 2025
The U.S. Food and Drug Administration (FDA) has approved Uplizna (inebilizumab-cdon) for the treatment of adults with immunoglobulin G4-related disease (IgG4-RD). IgG4-RD IgG4-RD is an immune-mediated condition that can affect multiple organ systems. Common...
by Madaline Spencer | Apr 9, 2025
This study evaluates the characterization of this atypical phenotype and focuses on 75 patients with ADPKD-like phenotypes with 41 different monoallelic predicted loss-of-function IFT140 variants. Autosomal dominant polycystic kidney disease (ADPKD) is caused by gene...
