New ERT for Pompe Disease As Effective as Older ERT

  Priya Kishnani, MD, Professor of Pediatrics at the Duke University School of Medicine, discusses the latest results from the COMET study assessing the efficacy of a new enzyme replacement therapy (ERT) to treat late-onset Pompe disease. Pompe disease is a...

More Research Needed To Understand Hypothalamic Obesity

  Chelsea Johnson, mother of a boy with hypothalamic obesity (HO), discusses the lack of understanding about this disease. HO is a rare endocrine disorder characterized by severe and debilitating obesity. Signs and symptoms of hypothalamic obesity include...

Two Siblings with SMA Receive Gene Therapy

   Emily Holtvluwer discusses the gene therapy her two children with spinal muscular atrophy (SMA) have received.   As Ms. Hotvluwar explains, her oldest child was diagnosed several months after symptoms began to appear while her second child was diagnosed in...

Gene Therapy and Sanfilippo Syndrome Type A

  Michael Hocquemiller, PhD, and Samantha Parker of Lysogene talk about the latest developments in the company’s gene therapy, LYS-SAF302, to treat Sanfilippo syndrome type A. Sanfilippo syndrome type A (MPS IIIA) is a progressive, life-threatening, and rare...