by Madaline Spencer | Apr 10, 2024
John Charlson, MD, Medical College of Wisconsin, discusses synovial sarcomas and the SPEARHEAD-1 clinical trial testing the safety and efficacy of afami-cel in this rare disease. Synovial sarcoma is a cancer that can originate from different types of...
by Madaline Spencer | Apr 8, 2024
Eosinophilic esophagitis (EoE) is a chronic autoimmune and digestive disorder in which large numbers of eosinophils, a type of white blood cell, are present in the esophagus. Eosinophils play a role in immune system regulation and fighting infections. Their...
by Madaline Spencer | Apr 8, 2024
Julia Alton, Executive Director at the Canadian Fabry Association, discusses the organization’s mission and their initiatives. Fabry disease is a lysosomal storage disease characterized by insufficient alpha-galactosidase (alpha-GAL). The deficiency of...
by Madaline Spencer | Apr 4, 2024
The U.S. Food and Drug Administration (FDA) has recently awarded Rare Pediatric Disease Designation for TNX-2900 (intranasal potentiated oxytocin) for the treatment of Prader-Willi Syndrome (PWS). PWS is a genetic condition caused by missing or...
by Madaline Spencer | Apr 4, 2024
Petra Oliva, PhD, Archimed Life Science, discusses newborn screening for metachromatic leukodystrophy (MLD). MLD is a genetic disorder caused by mutations in the ARSA or PSAP genes, which result in a deficiency of the enzyme arylsulfatase A and a...
