Anna's Story of Hope and Help: FUS-ALS with Sonja Kämpfer and Dr. Neil Shneider

Patient Empowerment Program: A Rare Disease Podcast

Stan Crooke speaks with Sonja Kampfer and Dr. Neil Shneider, associate professor of motor neuron disorders at Columbia Medical School, about Sonja’s daughter, Anna. Anna was diagnosed with an aggressive, fatal form of ALS at the age of 16. ALS is rare in teenagers, and this form of ALS is the worst of the worst. Sonja tells Anna’s story initial symptom onset, to diagnosis, and to Anna's response to ASO treatment. As a parent, Sonja reveals her full range of emotions during the duration of her daughter’s disease: puzzlement, pain, humility, and now optimism.

On This Episode We Discuss:

• Anna’s life before her symptoms appeared
• The road to a diagnosis and treatment– from Germany to New York City
• Neil Shneider and his work with ASOs
• The struggles of a family fighting a nano-rare disorder
• Anna’s mutation
• Anna’s remarkable progress