Sickle Cell Disease and Gene Therapy — Patient and Physician Perspectives | NEJM

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In this short documentary video from the _New England Journal of Medicine_ (NEJM), patients and physicians partner both to highlight the experience of living with sickle cell disease and to discuss the pathophysiology of the disease and new treatment strategies, including gene therapy.

Patients share their own stories of their interactions with the health care system and explore the challenging topics of racial disparity and health equity. Physicians express a cautious optimism as they review the risks and benefits of gene therapy.

0:00 Impact on Patients
2:20 Pathophysiology, Gene Therapy, and Disease Burden
5:38 Health Equity and History of Treatment
8:20 Gene Therapy Risk-Benefit

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I am Woman, educator, wife, mother, sister, daughter and a Sickle Cell warrior. I am SO happy to see such great information full Of real accounts and full of hope. I would Love if I could share this with my followers and students. Thank you so much for all of your hard work!! (I’m literally in tears after watching it because
It’s almost all my words… the words I would use to describe this Debilitating disease)

elizabethcifuentes
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"Strength perceived as weakness" Yes!

melissaware
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Outstanding documentary which depicted the perspective of the physicians approach to cure

awwalinuwagambo
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I absolutely love that this is your first topic of this new series! On 9/28/22's podcast, you mentioned you were looking for feedback. May I suggest that this be linked to CME/MOC credits in some fashion for us practicing physicians, especially subscribers to NEJM?

Vaitb
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I'm a huge fan of Dr. Thompson, she is amazing and inspiring! Great story, I share with bio students. 🥰

MsTlali
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This is a nice piece, but it feels like the racial bias component is an afterthought. That itself points to a problem with traditional medical minds who value the science of gene therapy over interactions with the people who might receive it. Both components are crucial to the delivery of high-quality care.

fredkron
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Gene therapy "LOVO-CEL" by Bluebird Bio

ahmedahm