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...
by Madaline Spencer | Jun 9, 2025
Pangkong Fox, PhD, Science Engagement Director at the CACNA1A Foundation and rare disease mom, discusses patient advocacy in CACNA1A-related disorders. CACNA1A-related disorders are a group of rare genetic neurological disorders caused by mutations in...
by Madaline Spencer | Jun 6, 2025
Matt Winton, PhD, Senior Vice President and Chief Operating Officer of Inozyme Pharma, discusses results from an analysis characterizing disease severity and progression in patients with ENPP1 deficiency. ENPP1 deficiency is a rare metabolic disease...
