Welcome to the Empowered Patient Podcast with Karen Jagoda. This show is a window into the latest innovations in applying generative AI, novel therapeutics and vaccines, and the changing dynamics in the medical and healthcare environment. One focus is on how providers, pharmaceutical companies, and payers are empowering patients. In addition, conversations are often about how providers, care facilities, pharmaceutical companies, and payers are being empowered by technology to improve patient outcomes and reduce friction across the healthcare landscape.
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- Virtual and digital health
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- Vaccines
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The audience includes life science leaders, researchers, medical professionals, patient advocates, digital health entrepreneurs, patients, caregivers, healthcare solution providers, students, journalists, and investors.
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Accelerating Development of Treatments for Spinal Muscular Atrophy with Karen Chen SMA Foundation TRANSCRIPT
Download PDFAug 30, 2021
Karen Chen is the Chief Executive Officer of the Spinal Muscular Atrophy Foundation. With a background in drug development having worked in pharma and biotech in neurodegenerative diseases like Alzheimer's and Parkinson's, Karen brings a deep understanding of the value of treating rare diseases that are genetically defined.
Karen explains, "The foundation is a little unusual as far as a nonprofit because we are really focused on our sole mission, which is to accelerate the development of treatments for SMA. And unlike other organizations, we're really not focused on raising awareness or raising funds as much as some of our sister SMA nonprofits. So we have really partnered with biotech and pharmaceutical companies to develop drugs for SMA. And SMA is one of the leading genetic causes of death in young children. It's actually the leading genetic killer of children."
"So even though there are three types, four if you include the adult form of SMA, it's all caused by the same underlying missing gene. The differences in severity are really a result of this backup gene that I talked about, SMN2."
#SMA #SpinalMuscularAtrophy #RareDisease #SMN2 #Evrysdi #Spinraza #Genentech #Biogen #SMAAwarenessMonth
