IC-MPGN and C3G Overview

Andrew Bomback, MD, Columbia University Medical Center, gives an IC-MPGN and C3G overview, two rare inflammatory kidney diseases.        Transcription: My name is Andrew Bomback. I am a nephrologist at Columbia University Irving Medical Center, where I...

How Is CIDP Diagnosed?

Richard Lewis, MD, Department of Neurology at Cedars-Sinai Medical Center, discusses diagnosing CIDP as well as the revised guidelines used to manage this rare disease.     Transcription:  There have been guidelines created over the years. Because it’s...

Genetic Testing to Diagnose Rare Diseases

Christine Eng, MD, Chief Medical Officer at Baylor Genetics, discusses whole genome sequencing and other forms of genetic testing in diagnosing rare diseases.       Transcription: All of the testing that we do really is designed to address unmet patient...

Osteogenesis Imperfecta Orbit Study

Eric Crombez, MD, Chief Medical Officer of Ultragenyx, discusses the latest results from the Phase 2/3 Orbit Study targeting osteogenesis imperfecta.     Transcription: Orbit is the name we have given to our ongoing phase 2/3 study. We have been talking...

Testing LUM-201 to Treat PGHD

Rick Hawkins, Chief Executive Officer at Lumos Pharma, discusses the OraGrowth212 Clinical Trial testing LUM-201 (ibutamoren) to treat pediatric growth hormone deficiency (PGHD).       Transcription: We have a global trial up and running. There are 80...