by James Radke | Jan 18, 2021
James Underberg, MD, Clinical Assistant Professor of Medicine at NYU Medical School, reviews the treatment options for homozygous familial hypercholesterolemia (HoFH). HoFH is a rare genetic disorder that leads to extremely high levels of cholesterol (i.e.,...
by James Radke | Oct 6, 2020
Swati Sathe, MD, of the CHDI Foundation addresses some of the unique issues faced by the rare disease community in the face of a world-wide pandemic. This presentation was part of the live GRIDS 2020-Virtual Summit held July 1, 2020 and is now available...
by James Radke | Sep 29, 2020
Shoshana Revel-Vilk, MD, Shaare Zedek Medical Center, Israel talks about how her team has used telehealth to assist patients with lysosomal storage disorders, such as Gaucher disease, Pompe disease, and Fabry disease, receive home-based care. This presentation...
by James Radke | Sep 21, 2020
Ari Zimran, MD, of the Shaare Zedek Medical Center, Israel talks about the impact the COVID-19 pandemic has had on treating patients with lysosomal storage disorders that require enzyme replacement therapies. This presentation was part of the live GRIDS...
by James Radke | Sep 17, 2020
Swati Sathe, MD, Medical Director of the CHDI Management/CHDI Foundation, presents an extensive report of Enroll-HD, a global dataset focused on Huntington’s disease (HD) that includes over 20,000 participants. Enroll-HD, as Dr. Sathe explains, is a...