The Balancing Act Explores Rare Diseases
February 28, 2019
Opening Up About Rare Diseases
On this special edition of The Balancing Act on Rare Disease Day, we are taking a look at the lives of patients who suffer from lesser-known conditions. In fact, approximately one in 10 Americans battle a rare disease. In the U.S., a disease is considered rare if it affects fewer than 200,000 people. If you’re struggling, know you are not alone.
First up, understanding Pheochromocytoma and Paraganglioma: rare neuroendocrine tumors that can dramatically impact survival in people of all ages.
Next, we’re learning about Pompe disease, a rare neuromuscular disorder affecting the hips, thighs, and shoulders, as well as muscles in the lungs. Sarah Gonzalez, head of medical diagnostics at Sanofi Genzyme, discusses how genetic testing plays an important role in obtaining a diagnosis.
Then, The Balancing Act welcomes back Ilana Jacqueline to discuss her brave journey as she continues battling a rare disease. Not only is she living, she’s thriving–but the fight has not been easy.
Clinical trials are an opportunity for patients to receive promising new therapies, but they can be difficult to navigate. Learn how The Leukemia & Lymphoma Society’s Clinical Trial Support Center is helping guide patients.
There are approximately 7,000 known rare diseases, but only 5% of them have an approved treatment. Learn how early intervention, proper management strategies, and clinical trials are key for rare disease patients.
Friedreich’s Ataxia is a progressive and debilitating neurological disorder. It's important to recognize symptoms and obtain an early diagnosis to properly manage the condition.