Current Status Not Enrolled Price Free Get Started Login to Enroll Course Resources Information TTR Amyloidosis: What Clinicians Need to Know About Its Pathophysiology Program: TTR Amyloidosis Slides Managing TTR Amyloidosis Module 1 slides Resources Koike H, Katsuno M. Ultrastructure in transthyretin amyloidosis: from pathophysiology to therapeutic insights. Biomedicines 2019;7:11 Hawkins PN,…
Kurt Gunter, MD, Inozyme Pharma, discusses data presented on ENPP1 deficiency and ABCC6 deficiency at ASBMR 2024.
Bruce Cree, MD, PhD, discusses a post hoc analysis of patient pain and quality of life data in treatment with Uplizna for NMO.
Andra Stratton, President and Co-founder of Lipodystrophy United, discusses the challenges of lipodystrophy from a patient’s perspective.
Lisa Berry, Genetic Counselor at Cincinnati Children’s Hospital, discusses results from Fabry patient survey.
Paul Bolno and Anne-Marie Li-Kwai-Cheung of Wave Life Sciences, discuss interim data from the FORWARD-53 trial for DMD.
John Carmichael, MD, discusses an analysis of growth hormone replacement therapy in patients under the age of 60 years.
Yuliya Linhares, MD, Chief of Lymphoma Services at Miami Cancer Institute, discusses strategies for shortening the diagnostic journey of CTCL.
Fadi Fakhouri, MD, PhD, discusses the results from a phase 2 trial of pegcetacoplan in patients with C3G and IC-MPGN.
Skand Shekhar, MD, Principal Investigator at the NIH, discusses Erdheim-Chester disease diagnosis and treatment options.
