by Madaline Spencer | Oct 3, 2024
Kurt Gunter, MD, Senior Vice President and Chief Medical Officer at Inozyme Pharma, discusses data presented on ENPP1 deficiency and ABCC6 deficiency at the 2024 American Society for Bone and Mineral Research meeting. ENPP1 ENPP1 deficiency is a rare...
by Madaline Spencer | Sep 30, 2024
Tiffany Chow, MD, Behavioral Neurologist, Vice President of Clinical Development at Alector, discusses a survey conducted to better understand caregivers’ experiences With frontotemporal dementia (FTD). FTDs are a group of neurodegenerative disorders...
by Madaline Spencer | Sep 19, 2024
Liz Marfia-Ash, President and Founder of GRIN2B Foundation, discusses life as a parent of a GRIN2B patient and resources her foundation offers. GRIN-related neurodevelopmental disorders are a family of rare genetic disorders caused by pathogenic...
by Madaline Spencer | Aug 27, 2024
Anita Longoria, a patient advocate, discusses her journey to a myasthenia gravis (MG) diagnosis. Myasthenia gravis (MG) is a chronic autoimmune neuromuscular disease characterized by weakness of the skeletal muscles. Common symptoms include weakness of...
by Madaline Spencer | Aug 15, 2024
Erdheim-Chester disease (ECD) is a rare condition affecting many parts of the body. Although diagnosed in some children, it most commonly affects adults. ECD causes the overproduction of histiocytes, which then accumulate in tissues and organs. Parts of the body that...
