by Madaline Spencer | Jun 13, 2025
Robert Mitchell-Thain, chair of the PBC Foundation, discusses how linerixibat reduces itching in patients with primary biliary cholangitis (PBC). PBC is a chronic, progressive autoimmune liver disease in which the bile ducts become inflamed and damaged....
by Madaline Spencer | Jun 12, 2025
Sumira Riaz, PhD, Health Psychologist & Patient Engagement Consultant at Unboxed Psychology, discusses the role of mental health in rare disease patient outcomes. Unboxed Psychology is an organization focused on improving the mental health and...
by Madaline Spencer | Jun 11, 2025
The U.S. Food and Drug Administration (FDA) has granted Fast Track designation to mavorixafor for the treatment of chronic neutropenia. Neutropenia is a rare genetic, autoimmune condition characterized by low levels of granulocytes, specifically neutrophils. The...
by Madaline Spencer | Jun 11, 2025
Jean Swidler, Executive Director for End The Legacy, discusses current challenges facing the amyotrophic lateral sclerosis (ALS) and frontotemporal dementias (FTD) communities. ALS is a motor neuron disease that leads to problems with muscle control and...
by Madaline Spencer | Jun 10, 2025
Nate Milam II, Patient Advocate for the Histiocytosis Association, discusses his diagnostic odyssey with histiocytosis. Histiocytosis is a rare hematologic disorder characterized by the overproduction of histiocytes, a type of white blood cell. In people...
