by Craig McCabe | Jun 3, 2024
P.J. Brooks, PhD, Acting Director of the Office of Rare Diseases Research at the National Center for Advancing Translational Sciences (NCATS), describes the Bespoke Gene Therapy Consortium (BGTC). The BGTC program is a private-public initiative designed...
by Craig McCabe | Jun 6, 2023
Jack Johnson, Executive Director of the Fabry Support and Information Group (FSIG) discusses the many research advances being made in Fabry disease. Fabry disease is a rare, X-linked lysosomal storage disorder. It is caused by the deficiency of the enzyme...
by Craig McCabe and James Radke, PhD | Jun 5, 2023
Rakesh Jain, MD, MPH, Professor, Department of Psychiatry, Texas Tech University School of Medicine – Permian Basin, discusses the recent Huntington’s disease treatment approval. Newly approved deutetrabenazine (Austedo XR) extended-release tablets...
by Craig McCabe and James Radke, PhD | Jun 1, 2023
Jack Johnson, of the Fabry Support and Information Group (FSIG) talks about the different types of pain often associated with Fabry disease. Fabry disease is a rare, X-linked lysosomal storage disorder that is caused by the deficiency of the enzyme...
by Craig McCabe | May 15, 2023
Jonathan Barratt, PhD, FRCP, Professor of Renal Medicine, University of Leicester, UK, describes the latest results of a phase 2b trial examining atacicept to treat immunoglobulin A (IgA) nephropathy. IgA nephropathy is a rare kidney disease characterized by...
