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...
by Madaline Spencer | Sep 18, 2024
A new gene therapy for X-linked retinoschisis (XLRS) has received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA). This rare genetic disorder affects the retina, causing vision loss primarily in males. The designation marks a crucial step...
