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EPISODE 06 || One Gene Therapy Shot = Cholesterol Control? || Medical media tech ||
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An experimental gene therapy has been found to slow the progression of Huntington’s disease, according to drugmaker uniQure, a major step towards the first genetic treatment for the genetic and fatal condition.
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The FDA has approved the first-ever medical treatment that allows deaf children with a rare genetic condition to hear. Regeneron says it will offer the new gene therapy free of charge to the small pool of eligible patients. David Muir reports.
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This is something that is close to me because I have sickle cell trait, but that doesn’t come with any of the things that sickle cell anemia comes with. I’m very lucky but now there’s hope for anyone who deals with this. Don’t forget T boz from TLC has sickle cell. Prodigy from my deep diet of sickle cell. It’s just a crazy thing to have #s#sicklecellm#manningschildrenshospitals#sicklecellawareness
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Two days ago the FDA approved the first gene therapy for people ages 2 & older for my disease, Spinal Muscular Atrophy, Itvisma. This is huge. Groundbreaking. Life changing. This gene therapy was approved in a slightly different form for babies born with SMA 2 & younger in 2019. I’ve been digesting this news for the past two days, reading more information & thoughts from other adults with SMA. I have previously been on Spinraza and I am currently taking a Evrysdi (a daily oral medication). Both
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