by Madaline Spencer | Mar 25, 2025
The U.S. Food and Drug Administration has approved Fabhalta (iptacopan) for the treatment of C3 glomerulopathy (C3G) in adults. C3G is a rare kidney disease characterized by damage to kidney glomeruli due to abnormal activation of the complement system. When C3...
by Madaline Spencer | Mar 25, 2025
Savara Therapeutics has announced a new diagnostic test for autoimmune pulmonary alveolar proteinosis (aPAP). aPAP is a rare autoimmune lung disorder. It is the most common form of pulmonary alveolar proteinosis. There is a wide variance in disease...
by Madaline Spencer | Mar 24, 2025
The U.S. Food and Drug Administration has approved Amvuttra (vutrisiran) for the treatment of transthyretin amyloid cardiomyopathy (ATTR-CM) in adults. ATTR-CM is a rare amyloidosis caused by aging or genetic mutations. This results in the misfolding of TTR protein...
by Madaline Spencer | Mar 24, 2025
Alessandra Maleddu, MD, Sarcoma Specialist at the University of Colorado, discusses the misdiagnosis of desmoid tumors and provides some resources for patients. Desmoid tumors are rare, benign, locally invasive, soft tissue tumors. They are commonly...
by Madaline Spencer | Mar 20, 2025
Bruce Leuchter, MD, Co-founder and CEO of Neurvati Neurosciences and GRIN Therapeutics, discusses the Breakthrough Therapy designation for radiprodil for treatment of patients with GRIN-related neurodevelopmental disorders. GRIN-related...
