Pediatrics

Guest: Jonathan Strober, MD Misinterpreted symptoms and limited access to treatment can make it difficult to manage myasthenia gravis among adolescents and young adults. Join Dr. Jonathan Strober as he explains how we can address gaps in care for this population. Dr. Strober is a Professor of Neurology at the University of California, San Francisco and the Director of the Neu

Guest: John F. Brandsema, MD As patients with Duchenne muscular dystrophy (DMD) age, their treatment regimens, considerations, and goals change. That’s why Dr. John Brandsema is here to talk about how we can best treat and support patients with DMD throughout their entire care journey. Dr. Brandsema is the Neuromuscular Section Head at the Children’s Hospital of Philadelp

Guest: John F. Brandsema, MD Between 60 and 70 percent of patients with Duchenne muscular dystrophy (DMD) also have a diagnosable neurobehavioral phenotype like autism, ADHD, and anxiety. Here to talk about these common neurobehavioral issues in patients with DMD and how we can manage them is Dr. John Brandsema, Neuromuscular Section Head at the Children’s Hospital of Phila

Host: Charles Turck, PharmD, BCPS, BCCCP Guest: John F. Brandsema, MD The gene therapy delandistrogene moxeparvovec was recently approved for patients with Duchenne muscular dystrophy (DMD) who are at least 4 years old. This expanded approval is based on the efficacy and safety results from the EMBARK trial. Joining Dr. Charles Turck to break down those key findings and th

Host: Charles Turck, PharmD, BCPS, BCCCP Guest: Nadia Merchant, MD Guest: Melody Shi, MD Guest: Despoina Galetaki, MD Steroids are one of the main treatments for patients with Duchenne muscular dystrophy (DMD), but prolonged steroid use can come with many endocrine side effects, like weight gain, insulin resistance, and adrenal insufficiency. To learn more about thes

Host: Jody Takemoto, PhD Guest: Donald Y. Leung, MD, PhD There’s currently no way of knowing who’s going to develop a food allergy and who isn’t, which means early treatment or even prevention can't begin. But that might all change based on a recent study, which found that skin biomarkers may be able to predict the development of food allergies in pediatric patients.

Host: Jacob R. Bledsoe, MD WHIM syndrome is a rare, inherited, combined immunodeficiency disease caused by reduced mobilization and trafficking of white blood cells from the bone marrow due to over-signaling of the CXCR4/CXCL12 pathway. WHIM syndrome is named for its four common clinical findings. The diagnostic challenges arise because not all symptoms are required for a dia

Host: Peter E. Newburger, MD WHIM syndrome is a rare, inherited, combined immunodeficiency disease caused by reduced mobilization and trafficking of white blood cells from the bone marrow due to over-signaling of the CXCR4/CXCL12 pathway. WHIM syndrome is named for its four common clinical findings. The diagnostic challenges arise because not all symptoms are required for a d

Host: Teresa K. Tarrant, MD WHIM syndrome is a rare, inherited, combined immunodeficiency disease caused by reduced mobilization and trafficking of white blood cells from the bone marrow due to over-signaling of the CXCR4/CXCL12 pathway. WHIM syndrome is named for its four common clinical findings. The diagnostic challenges arise because not all symptoms are required for a di

Host: Peter E. Newburger, MD WHIM syndrome is a rare, inherited, combined immunodeficiency disease caused by reduced mobilization and trafficking of white blood cells from the bone marrow due to over-signaling of the CXCR4/CXCL12 pathway. WHIM syndrome is named for its four common clinical findings. The diagnostic challenges arise because not all symptoms are required for a d