SMA Type 1: How Gene Therapy Works

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.

[bishaljha]

HELLO TO People watching from SNT & TECH COURSE BY SHIVIN

[faye]

My daughter, born 9 days ago and diagnosed SMA1, gets this treatment next week!!

[nusaibamohammed]

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.

[Professorofeverythingvlogs]

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.

[manishagurung]

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.

[zedroscrid]

Hoping to get approved for this Monday for my daughter.

[moosha]

The idea that a life could be ruined because they don't have money to buy the treatment is terrifying

[larryprice]

When you're sick all you want to be is cured we're willing to take the chance

[nandneesingh]

May god help all those who are suffering from this disease 😥😢😭🙏 and also to my country india

[jennysmalls]

I just received my son’s newborn screening results and SMN1 exon 7 present

[MAZe]

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?

[dukukoirala]

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

[shilpaskitchen]

Hiii is this usefull for cerebral palsy ??

[SmilewithTanu-___Tanu-Nigam]

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

[Vlllllllla]

Perfectly understandable by this video. Thx!

[larryprice]

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

[hmaraislampiyaraislam]

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

[ethanfernandez]

what therapy is this exactly? Spinraza or new AVXS-101?

[serajoe]

How much can this treatment cost? I'm tired of seeing my child in pains. Please what's the cost

[Aalasttal]

Great topic, unfortunately the drug act for gene therapy for SMA 1 disease ( ZOLGENSMA) its cost equal 2.1 M dollars!