by Peter Ciszewski | Mar 3, 2022
Giles Campion, MD, Head of R&D at Silence Therapeutics, discuss SLN124, a gene silencing therapy in development for the treatment of thalassemia. As Dr. Campion explains, thalassemia is an inherited blood disorder that reduces the production of functional...
by Peter Ciszewski | Mar 1, 2022
Paula Ragan, PhD, CEO and President, X4 Pharmaceuticals, discusses data presented at The American Society of Hematology Meeting & Exposition (ASH 2021) on a phase 2 open-label extension study of mavorixafor for the treatment of WHIM syndrome. WHIM syndrome...
by Peter Ciszewski | Mar 1, 2022
Data sharing is a common request in the rare disease community. Unfortunately, the realities of our current research infrastructure make it problematic for researchers to share their data. The net result is a plethora of data silos that can dramatically delay...
by Peter Ciszewski | Feb 28, 2022
David Kronn, MD, Associate Professor of Pathology and Pediatrics at New York Medical College, provides an overview of the CME program that he and Jerry Vockley, MD, PhD, Chief of Genetic and Genomic Medicine at the University of Pittsburgh are hosting that...
by Peter Ciszewski | Feb 28, 2022
Jerry Vockley, MD, PhD, Head of the Division of Medical Genetics at UPMC Children’s Hospital of Pittsburgh, gives an update on the phase 2 trial testing SYNB1618 to treat phenylketonuria (PKU). PKU is a rare genetic metabolic disorder that results in reduced...
