by Madaline Spencer | Jun 4, 2024
David Gusick, Founder of Somebody To Talk To, discusses mental health in patients with rare diseases. Somebody To Talk To focuses on providing mental health resources and support for patients, especially those with Duchenne muscular dystrophy. Through...
by Madaline Spencer | Jun 3, 2024
Cathleen Lutz, PhD, Vice President of the Rare Disease Translational Center at The Jackson Laboratory (JAX), discusses how the laboratory is addressing unmet needs in rare diseases. JAX aims to understand the underlying pathophysiologies of diseases and...
by Madaline Spencer | May 31, 2024
Amy Gray, Chief Executive Officer of the Undiagnosed Diseases Network Foundation (UDNF), discusses the organization and what they do for patients of undiagnosed rare diseases. As Ms. Gray explains, the journey to a diagnosis can be a long and...
by Madaline Spencer | May 30, 2024
The U.S. Food and Drug Administration (FDA) has approved Bkemv (eculizumab-aeeb), an interchangeable biosimilar to Soliris (eculizumab). The treatment is approved for both paroxysmal nocturnal hemoglobinuria (PNH), to reduce hemolysis, and atypical hemolytic uremic...
by Madaline Spencer | May 29, 2024
Amanda Cali, Co-founder of Tin Soldiers Global, discusses how the organization helps find patients with fibrodysplasia ossificans progressiva (FOP) and other rare diseases. FOP is a rare disorder in which skeletal muscle and connective tissue are...
