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Podcast: It Happened To Me: A Rare Disease and Medical Challenges Podcast
Episode: #51 Niemann-Pick Type C: Understanding Symptoms, Genetics, and New Treatments
Description: In this episode of It Happened To Me, we dive into the world of Niemann-Pick Type C (NPC), a rare genetic condition that affects fewer than 1,000 people in the United States. NPC is a progressive disorder impacting the brain, nerves, and major organs, often referred to as "childhood Alzheimer’s" due to its neurological effects.
Our guests bring both personal and professional expertise to the conversation:
Barbara Lazarus is the mother of two adult sons (now in their 30s), who have Niemann-Pick Type C disease. David is taking Myplyffa, through an expanded access program. Both David and...