Real-World Cases in Pediatric Low-Grade Glioma: Exploring Targeted Therapies
Alexandria May, PharmD, BCPS
Angela Waanders, MD, MPH, MS
Ashley S. Margol, MD, MS
Improving Survival and Function in Pediatric TK2d with Nucleoside Therapy
Charles Turck, PharmD, BCPS, BCCCP
Caterina Garone, MD, PhD
Uncovering the Disease Burden of Untreated Pediatric TK2 Deficiency
Cristina Domínguez González, MD, PhD
Optimizing the Role for Corticosteroids Within the Evolving DMD Treatment Landscape
Stephen Chrzanowski, MD, PhD
Aravindhan Veerapandiyan, MD
Genetic Testing in Pediatrics: Identifying Neurodevelopmental Disorders Early
Jennifer M. Kalish, MD, PhD, FAAP
Addressing Barriers to MenB and MenACWY Vaccination
Evaluating MenB Vaccines Beyond the Traditional hSBA Assay
Pregnancy and Infant Outcomes in Multiple Sclerosis: Findings from the MAPLE-MS Pharmacovigilance Program
ASD in Children with SMA Type I: Diagnostic Challenges in a Multifactorial Context
Safety and Efficacy of Onasemnogene Abeparvovec in Pediatric SMA Across Expanded Weight Cohorts
Reevaluating Spinal Muscular Atrophy Therapy: Signs, Tools, and Strategies
Brian P. McDonough, MD, FAAFP
Nancy Kuntz, MD
Exploring Circulating CD34+ Cells as a Biomarker of Fracture Risk in DMD
Quantifying the Hidden Cost Burden of Duchenne Muscular Dystrophy on U.S. Households
Cardiopulmonary Exercise Testing Sheds Light on Early Physiologic Decline in Duchenne Muscular Dystrophy
Treatment of a Broader Population of Patients With DMD
Crystal Proud, MD
Erika Finanger, MD, MS
Spinal Muscular Atrophy Care: Optimizing Therapeutic Decision Making
John Brandsema, MD
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