Several recently published studies may help enhance our treatment of spinal muscular atrophy (SMA). Review key points of some of them below. Evaluating a Novel Myostatin Inhibitor as an SMA Treatment A new article in Advances in Therapy describes a phase 1 trial of a monoclonal antibody intervention being developed as a treatment for SMA1.…
There have been several recent publications related to spinal muscular atrophy (SMA). Below is a summary of some pieces that add to our understanding of the disease. Identifying Modifiers of SMN Proteins A study out of Bethesda, Maryland, published in Cell Reports, aimed at investigating the mechanisms by which survival motor neuron (SMN) proteins are…
Although we are making headway in understanding and treating people living with Alagille Syndrome (ALGS) — a rare genetic disorder that manifests in a variety of ways — several questions remain. Here, Henry C. Lin, MD, pediatric gastroenterologist in the Division of Gastroenterology at Doernbecher Children’s Hospital, Oregon Health & Science University, shares his perspective…
Several pieces of recently published research may aid clinicians in managing spinal muscular atrophy (SMA) across the life span. Review highlights of some of the studies below. Early Treatment for Preterm Infants With SMA SMA is being diagnosed earlier than ever before, even before symptoms arise. For preterm infants who do not yet demonstrate SMA…
