by Madaline Spencer | Sep 26, 2024
Andrew Dauber, MD, Chief of Endocrinology at Children’s National Hospital, discusses data presented on the efficacy of vosoritide in genetic skeletal conditions. Disease Overview Achondroplasia is a rare skeletal disorder that prevents the changing of cartilage...
by Madaline Spencer | Sep 25, 2024
Shubh Goel, VP, Head of Immuno-Oncology and GI Tumors Franchise for US Oncology at AstraZeneca, discusses results from the HIMALAYA clinical trial for hepatocellular carcinoma (HCC). HCC is a rare liver carcinoma characterized by nodules occurring within...
by Madaline Spencer | Sep 24, 2024
Elizabeth Berry-Kravis, MD, PhD, Pediatric Neurologist at Rush University Medical Center, discusses the development of an investigational drug for Fragile X syndrome (FXS). FXS is a rare genetic condition involving mutations in part of the X chromosome....
by Madaline Spencer | Sep 23, 2024
The US Food and Drug Administration has approved Miplyffa (arimoclomol) for the treatment of Niemann Pick type C (NPC), the first approved therapy for this indication. NPC is a rare lysosomal disorder characterized by variable clinical signs, depending on the age of...
by Madaline Spencer | Sep 19, 2024
Liz Marfia-Ash, President and Founder of GRIN2B Foundation, discusses life as a parent of a GRIN2B patient and resources her foundation offers. GRIN-related neurodevelopmental disorders are a family of rare genetic disorders caused by pathogenic...
