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

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.
The New England Journal of Medicine is the world’s leading general medical journal. Continuously published for over 200 years, NEJM publishes peer-reviewed research along with interactive clinical content for physicians, educators, and the global medical community at www.NEJM.org.
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
#sicklecelldisease

[@elizabethcifuentes715]

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)

[@Smilingjewel24]

Please share!!!!

[@catherineorr7646]

@@Smilingjewel24 Yes!

[@NEJMgroup]

Thank you, Elizabeth, for your kind words about the video. Your feedback is greatly appreciated and we would be happy for you to share the video with those who would enjoy seeing it. Take good care and best wishes.

[@awwalinuwagambo6504]

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

[@melissaware4728]

"Strength perceived as weakness" Yes!

[@Vaitb85]

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?

[@NEJMgroup]

Thank you for your comment about the video and for taking time to provide us with your feedback. Unfortunately CME/MOC credit for watching these videos is not available at this time. It is an interesting suggestion and can be considered for future enhancements.

[@MsTlali]

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

[@ahmedahm1]

Gene therapy "LOVO-CEL" by Bluebird Bio