by Madaline Spencer | Aug 7, 2024
Jula Inrig, MD, Chief Medical Officer at Travere Therapeutics, discusses a clinical trial that tested sparsentan for treatment of IgA nephropathy, compared to traditional RAS inhibitors. IgA nephropathy (IgAN) is a rare kidney disorder that occurs when...
by Madaline Spencer | Aug 6, 2024
The U.S. Food and Drug Administration (FDA) Genetic Metabolic Diseases Advisory Committee voted in favor of the data supporting arimoclomol as an effective treatment for patients with Niemann-Pick disease type C (NPC). NPC is a rare lysosomal storage disease...
by Madaline Spencer | Aug 5, 2024
Stiff person syndrome (SPS) is a rare, progressive disease that affects the nervous system, specifically the brain and spinal cord. The syndrome affects twice as many women as men. SPS is caused by increased muscle activity due to decreased inhibition of the central...
by Madaline Spencer | Aug 3, 2024
The U.S. Food and Drug Administration (FDA) has approved Tecelra (afamitresgene autoleucel or afami-cel) for the treatment of unresectable or metastatic synovial sarcoma in adults with certain HLA types who have received prior chemotherapy. Synovial sarcoma is a rare...
by Madaline Spencer | Aug 2, 2024
John Carmichael, MD, Co-director of the USC Pituitary Center and Clinical Chief for the Division of Endocrinology, discusses an analysis of growth hormone replacement therapy in patients under the age of 60 years. Growth hormone deficiency (GHD) is a...
