by James Radke, PhD | Jun 14, 2022
The U.S. Food and Drug Administration (FDA) has approved of vutrisiran (Amvuttra) for the treatment of polyneuropathy observed in patients with hereditary ATTR (hATTR) amyloidosis. hATTR amyloidosis is an inherited and progressively debilitating disease caused by...
by James Radke, PhD | Jun 10, 2022
Sherif El-Harazi, MD, Ophthalmologist, and Medical Director and Founder of Lugene Eye Institute and Global Research Management, gives an overview of vernal keratoconjunctivitis (VKC). As Dr. El-Harazi explains, VKC is a rare and recurrent allergic eye...
by James Radke, PhD | Jun 9, 2022
Dean Suhr, President and Co-Founder of the MLD Foundation, discusses a paper and corresponding poster presentation on a recent survey of newborn screening experts on strategies to modernize newborn screening in the United States. The paper was published in JAMA...
by James Radke, PhD | May 23, 2022
Olaf Bodamer, MD, PhD, Associate Chief of Genetics and Genomics at Boston Children’s Hospital, discusses how the National Organization for Rare Disorders’ (NORD) Rare Disease Centers of Excellence addresses unmet needs of the rare disease community. There...
by James Radke, PhD | May 4, 2022
Gregory Grabowski, MD, Professor Emeritus at University of Cincinnati College of Medicine highlights the latest research about Gaucher disease presented at WORLDSymposium 2022. This free CME program provides expert analysis on the clinical relevance of this...
