by Peter Ciszewski | Nov 8, 2022
Mark Forman, MD, PhD, Chief Medical Officer at Passage Bio, provides an overview of GM1 gangliosidosis. As Dr. Forman explains, GM1 gangliosidosis is an inherited lysosomal storage disorder caused by mutations in the GLB1 gene, which encodes the lysosomal...
by Peter Ciszewski | Nov 7, 2022
Gerald Vockley, MD, PhD, Head of the Division of Medical Genetics at UPMC Children’s Hospital of Pittsburgh, discusses new data testing a probiotic to treat individuals with phenylketonuria (PKU). PKU is a rare genetic metabolic disorder that results in...
by Peter Ciszewski | Oct 31, 2022
Olaf Bodamer, MD, PhD, Associate Chief of Genetics and Genomics at Boston Children’s Hospital, discusses how the National Organization for Rare Disorders’ (NORD) Rare Disease Centers of Excellence may help rare disease patients receive the best care...
by Peter Ciszewski | Oct 26, 2022
Nathalie Dubois-Stringfellow, PhD, Chief Development Officer at Sangamo Therapeutics, discussed the promising results from their ongoing gene therapy trial to treat Fabry disease. Fabry disease is a rare X-linked lysosomal storage disorder that results in the...
by Peter Ciszewski | Oct 25, 2022
The U.S. Food and Drug Administration (FDA) has given accelerated approval to teclistamab-cqyv (Tecvayli) to treat adults with with relapsed or refractory multiple myeloma, who previously received four or more prior lines of therapy, including a proteasome inhibitor,...
