by James Radke, PhD | Jun 24, 2022
P.J. Brooks, PhD, Acting Director of the Office of Rare Diseases Research at the National Center for Advancing Translational Sciences (NCATS), describes NCATS’ push to increase efficiency and decrease costs in the development of rare disease treatments. As Dr....
by James Radke, PhD | Jun 17, 2022
The U.S. Food and Drug Administration has approved a supplemental indication for setmelanotide (Imcivree) for chronic weight management in patients ≥6 years of age with obesity due to Bardet-Biedl Syndrome (BBS). BBS is a rare genetic disease that impairs the hunger...
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...
