SMA Type 1: How Gene Therapy Works
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- Опубликовано: 7 окт 2024
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A one-time intravenous infusion of the high dose of gene therapy extended the survival of patients with spinal muscular atrophy type 1 (SMA1) in a Phase 1 clinical trial, according to a study published Nov. 1, 2017 in the New England Journal of Medicine. This animation describes how the process works.
HELLO TO People watching from SNT & TECH COURSE BY SHIVIN
Lol
😀hello
My daughter, born 9 days ago and diagnosed SMA1, gets this treatment next week!!
Am so proud with such a treatment and I hope my son whose also diagnosed with SMA1 could be treated with spinraza , he's 3 years now and we are suffering from the impact of the disease emotionally and financially and living in a developing country with such a disease is a struggle. Am always praying for him and for a chance of proper treatment. Please help us.
You need to talk about IT more, make Insta páge, post photo, contacts! Do it. !!
God help all this people who are suffering from this disease 😢🙏
@@oxioxi5 thanks for your support
Just pray to allah, the moon god
😭😭😭 i lost my niece she was 2 yr old on 26 may 2021
She suffered the same
Could not balance her neck
Could not roll... nothing like a normal baby 😭😭😭😭
Hoping to get approved for this Monday for my daughter.
Jon Ward Where did u sign up at needing this for my daughter please
Angela Philip you go to your childs neurologist and ask for treatment options for SMA and they will tell you spinraza or zolgensma (depending on where you are and what treatments are available there) they will submit a request to your insurance and then your insurance will either approve it or deny it. You can repeal a number of times if they say no. I hope you have success in getting this gene therapy good luck
My son has sma 1. He was treated with his first dose two weeks ago of spinraza, his second dose was this afternoon. He's a month old. One thing I noticed is he is moving more since his first treatment which is a blessing.. hoping to get him approved for zolgensma soon . I'll be praying for you and your family and hope the best for yall.
I hope you guys get approved!
Guys, one kid in Russia has SMA1 parents asking for help but I have no idea how help them. Please, if you have any kind of information, like clinik's site or doctor's phone, please live a comment or email me : segeimarkin811@gmail.com
PS. Sorry for my English.
Truly a marvel of scientific advancement. Those kids who were bound to die due to this neuro muscular defect, will now survive due to this miracle of science.
Why this treatment is such a expensive? I wish there would be some consideration, so many kids could get back their life. It's heart wrenching to know there is a treatment but parents have to watch their child dying because of unaffordable treatment cost.
Mri baby sma type 1 me thy uski death hogae 2018 me 7 month me
Unfortunately everything new is expensive :( Governments should support it because the company spent a hell lot of money for their research, production is also very expensive as it’s not possible to mass-produce this virus effectively so drug’s self cost is very high. Without being funded, the company will eventually went bankrupt and will not be able to fund further researches. I hope that eventually it will be possible to decrease the cost to help more children.
Any product on this planet is expensive in the initial period of its release
@@reema.solangi6251 hello di
Even i lost my niece 2 yr old on 26 may 2021...even she suffered the same
@@reema.solangi6251 aapko or baby hai kya
I just received my son’s newborn screening results and SMN1 exon 7 present
How is your baby now?
Perfectly understandable by this video. Thx!
Really
May god help all those who are suffering from this disease 😥😢😭🙏 and also to my country india
😭💔
I becker muscular dystrophy patient
@@godpinakyt may god help you 🙏😔 take of your self
17.5 crores is the price. Good luck arranging that. It's either you be lucky or be rich
recently a child from my town of West Bengal was suffering from SMA type 1 and he had administered ZOLGENSMA drug price@ 17.5 crore at AIIMS New Delhi and it is reported that he is showing improvement in his motor neuron action. God may save the little kid.
Interesting!
A M A Z I N G ! ! !
When you're sick all you want to be is cured we're willing to take the chance
The idea that a life could be ruined because they don't have money to buy the treatment is terrifying
Thanks❤
since it says it does not integrate with the native gene. how does it regulates to limit the synthesize of the gene without being in the chromosome? how does it works epigenetically?
I think, If you can make the proper gene for the synthesis of that specific protein for an essential cell (in the lab). It doesn't have to be in the chromosomes, since they already lack the wanted gene for that protein.
I am guessing its a plasmid because a double stranded piece of DNA that isnt circular is more prone to being eaten at the ends. Either that or it has some form of capping going on to protect the ends. A plasmid (circular bit of double stranded DNA) is being copied just like the entire genome is being copied during cell division. If it isnt copied, there are certain factors that mitigate the transcription and translation of that piece of DNA to make the protein needed for function. There are many more t herapies in the making. CRISPR/Cas9 is currently being thought of as a future editing technique where they basically cut out the defective gene, and together with the molecular scissors they also include the correct version of the gene that the cell can put in place of it. SCIENCE
Well, that's the thing with gene therapy. Researchers use preclinical models to assess feasibility, efficacy, safety and transgene expression. During dose escalation phases, researchers measure transgene expression to get an idea of what dosage is required for long-term expression. There are also multiple vectors in which they use to optimise gene expression too. Viral vector selection and optimisation is key to transgene expression.
Thank you virus. I am saying it at the worst outbreak month of COVID pandemic.
God bless us
hoping Usman and siyona good health. siyona got medicine and Usman haven't get. please pray for usman . it is 2 months he is suffering from it
Hiii is this usefull for cerebral palsy ??
My baby is 7 month old and he is suffering SMA . I am from India please help us 🙏 how can I take this treatment and what is the cost in this treatment
try to contact the company Biogen and then ask about the early access program!! good luck!
You need only one injection which name Zolgensma. It cost $ 2.347.750😕 To one baby from Ukraine, all country was collecting this money.
When they going to start using gene therapy to cure HIV I got the disease 34 years ago I was like 19 years old dying and desperate please help thank you
Great topic, unfortunately the drug act for gene therapy for SMA 1 disease ( ZOLGENSMA) its cost equal 2.1 M dollars!
,😶...
How much can this treatment cost? I'm tired of seeing my child in pains. Please what's the cost
2 million dollars.. really
Trouble in the highest order.
The genetic sequence that they used is public and so is their protocol.
@@creativityhub1350 Design of an airplane is also public info. Good luck with making one yourself.
@@kacperrucinski unless you're an aviation engineer who knows what he/she's doing and has complete access to the tools and materials. If somebody personally doesn't know how to do it then that's on them but it's not an impossibility.
Where do they get the SMN 1 gene?
Do it helps for Congenital Myasthenia?
No
Smile with Tanu
My so is 2 month old and he is suffering SMA . I am from Pakistan please help us 🙏 how can I take this treatment and what is the cost in this treatment
hi I'm amber! my daughters have sma type 1 and were treated and there is hope! try to get ahold of biogen and then ask about the early access program for zolgensma!
preparing body to the genes replacement.
what therapy is this exactly? Spinraza or new AVXS-101?
This is zolgensa
@@Shani249 Zolgensma*
@@ivelisseys7285 haha yes that
This treatment cost ₹16 crores ........why the hell it's so costly.
Lesser the demand of a medecine, more costly it becomes.
$ 2.000.000,00
is sma completely cured with this treatment.pls reply
hi my husband (this is his wife using his youtube) and I have 2 daughters who were treated at different times and had different outcomes. if given before symptoms start then yes they will be healthy and show no symptoms but if given after progression and muscle wasting has started it will help repair the muscles and keep what strength they do have.
try to contact Biogen and ask about the early access program! good luck!
This gene therapy are available in India??
No, and unlikely to be in the next few years.
in.mobile.reuters.com/article/amp/idINKBN1YN1EI
that is an article about access in india it was written in 2019 I'm not sure it will help but I hope it does
No... It's mainly cure treatment is with japan, America..... But not with India
@@nandneesingh3065 abhi usa me bhi nhi hai March last ya April me aane ki umeed ki ja sakti h
Publically offered it should be inexpensive or paid for insurance offered to vetenary clincs at lower price 250 per treatments reverse aging.
i m suffering from SPINAL MUSCULER ATROPHY TYPE ||| please help me.and is gene therapy cure me??
my blessings to u and u will cure soon , Universe is listening you
Thought this is reverse aging treatments
price this vaccine,
srry i was just kidding
💔
For people on here who can't afford it, the genetic sequence that Zolgensma used is public info. Their administration protocol as well.
Good luck with making the transgene in your garage.
@@kacperrucinski done. Not hard at all.
@@creativityhub1350 Can you make a youtube tutorial? Thank you.
@@creativityhub1350 so if it s something that you can even do yourself than why it costs 2 million dollars? Tell me the reason before it. By the way i m not trying to deny you i just wonder so answer my question whitout getting offended.
@@crewyard9917 because it takes companies years of research and cost for employees, profit margin considerations, etc.
this is some umbrella inc shit right here
Martin Mai iS tHiS sKyNeT
@Gabriel P It's a joke on ow people act like every new technological item is gonna kill them. I though the joke was simple enough.
is LIMB-GURDLE MD , SMA desease ?
No it's a different type of muscular dystrophy
omg how complex is our body structure😢