by Madaline Spencer and James Radke, PhD | May 11, 2023
Christian Marsolais, Ph.D., Chief Medical Officer of Theratechnologies, discusses three studies presented at the American Association for Cancer Research (AACR) conference targeting SORT1-positive cancers. Sudocetaxel zendusortide (TH1902) is a proprietary...
by James Radke, PhD | May 10, 2023
The U.S. Food and Drug Administration (FDA) has approved pegunigalsidase alfa (Elfabrio), a plant-based enzyme replacement therapy with an initial half-life of 78.9 ± 10.3 hours, for the treatment of adult patients with Fabry disease. Fabry disease is a rare X-linked...
by James Radke, PhD | May 9, 2023
Giacomo Chiesi, Head of Chiesi Global Rare Diseases, discusses velmanase alfa (Lamzede), which was recently approved by the U.S. Food and Drug Administration (FDA) for the treatment of alpha mannosidosis. Alpha mannosidosis is a multisystemic, lysosomal disorder...
by James Radke, PhD and Craig McCabe | May 8, 2023
Brenna Bentley , MS, GCG, genetic counselor and patient research coordinator for the United MSD Foundation lists some of the more common symptoms observed in children with multiple sulfatase deficiency (MSD). MSD is a rare genetic disease due to mutations in...
by Craig McCabe and James Radke, PhD | May 8, 2023
Gerard Vockley, MD, PhD, Professor of Pediatrics and Human Genetics at the University of Pittsburgh, provides an overview of galactosemia. Galactosemia is a rare, metabolic disorder that affects an individual’s ability to metabolize galactose, which is...
