FDA approves gene-editing therapy for sickle cell disease
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- Опубликовано: 5 авг 2024
- The FDA approved gene-editing therapy Casgevy to treat sickle cell disease. Developed by Vertex Pharmaceuticals (VRTX) and CRISPR Therapeutics (CRSP), the one-time treatment harnesses gene editing advancements.
Vertex CEO Dr. Reshma Kewalramani says the approach involves extracting a patient's stem cells, correcting the defect in those cells using CRISPR gene editing tools, then re-infusing the edited cells into the patient - relieving painful symptoms.
Patients who undergo this $2.2 million procedure may eliminate painful sickle cell crises called VOC's. Kewalramani contrasts the cost against the $4-6 million lifetime price tag for conventional sickle cell management.
#youtube #genetics #yahoofinance
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the treatment is worth it but unfortunately most of the people affected will not be able to afford $2.2 million
This is a big deal. As an older brother to a sister with sickle cell disease, and as a Physician who has treated many patients for vaso-occlusive pain crises, it is impossible to overstate how life-changing this will be for many people.
From the Casgevy Package insert:
"The most common Grade 3 or 4 laboratory abnormalities (≥ 50%) were
neutropenia, thrombocytopenia, leukopenia, **anemia**, and lymphopenia"
Good news
Setting that price tag is like swinging a piece of chicken and rubbing it against a dog's nose without ever intending the dog to take a bite - cold! :(
I don't know that sickle cell anaemia has been, historically, ignored. I have heard about it for years. And now it seems that this disease has been put at the top of the list for use with this therapy.
Whatever
We can't afford 😢
Really? She is trying to justify the 2.2 mil cost for this treatment for people suffering from this disease! Sad and disgusting! I understand they need to recuperate for their research…but 2.2mil?
I thought I heard wrong - 2m is definitely not feasible for the masses. Should this be called THE SICKLE CELL BREAKTHROUGH THERAPY FOR THE AFFLUENT? If this is to be perceived as a sincere scientific breakthrough, it should not have money at its core never mind it serving as a barrier :(
😂😂😂😂❤❤❤
Scary Frankenstein medicine
Why?
@kisstock Pharmaceuticals all have side effects. Pharmaceutical companies I'm more interested in profit and control. There are many cures for most diseases that do not involve Pharmaceuticals. Pharmaceuticals are only 100 years old. Look up Rockefeller medicine. Look up Kellogg and Rockefeller..
There’s nothing at all scary about it if you understand the science / technology.
It will save lives.
@@MehulPatelLXC I think he means he doesn't trust the people behind it.
A knife is a knife, but would you trust a chef with it or a serial killer?