New Gene Therapy Transforms SMA (Spinal Muscular Atrophy)

URMC has been tapped as one of the first institutions in the U.S. to offer a new gene replacement therapy to treat spinal muscular atrophy (SMA). Until a few years ago, an SMA diagnosis was tantamount to a death sentence. Learn how this cutting-edge therapy is transforming the lives of patients and their families.

[@teachrare]

I love the Easter Egg of including Wonder in the background while interviewing in the library!

[@jscarter0110]

People have no idea how much pain these child illnesses cause, thank goodness for the smart people developing/putting gene therapy into actual practice, it’s gonna do so many wonderful things…spread the word, we don’t need to fix dumb shit, we need to fix REAL problems like this!!

[@faisalmasood8217]

My daughter passed away due to this disease, She was only 8 months old I really miss her alot ...

[@emu6563]

Sorry to hear that😭 rest in peace little soul

[@ayeshasiddiqua4295]

I also lost my 3 months old daughter 2 months ago.. I can feel your pain💔

[@fatimasalman8921]

@faisalmasood8217 aap ki bytti ko kia hua tha? Kia wo bazu hilati thi kia ? Uski Kyse condition thi ? Myri bhtiji ki bhi yahi report karwai ha Dr ny

[@ruthdanielson919]

This treatment is a miracle, just wishing it could be used in older children, even if just to stop the progression of the disease. Seeing my nine year old patient have improved muscular activity would be wonderful. Seeing her talk and live withour a ventilator, awsome. Seeing her walk, talk, sing dance and play would be the answer to my prayers. Thank you, physicians and researchers for gene therapy. Thank you, thank you, thank you!!! And, most of all, that you, Heavenly Father!!!!!

[@Kevo7979]

In 2018, I had an expensive genetic test in Bangkok to check if I was a carrier (SMA among many diseases) when my wife was carrying my boy (20 weeks, I think). If my understanding is correct, SMA condition requires both parents to be the carriers, and even with that, it's 1-in-4 chance. Fortunately, I was not a carrier. I hope the development of this gene therapy will go a long way to help cure the disease or improve lives of children affected by it.

[@chilar6]

What about your wife? Was she a carrier ?

[@kayan_dev]

Where is this hospital ?

[@kattarhindu107]

I'm suffering from spino cerebellar ataxia... Is there any permanent treatment available for SCA

[@nishanakarmi8572]

is there any kind of charity that can help an infant recently diagnosed with SMA type 1? The treatment is very expensive.

[@MAD4FAK4]

This therapy costs $2.1M.. I'm not going to say anything else, I think you all get it.

[@adrianamirauta8206]

How do these people sleep at night? They are amazing scientists for discovering this vaccin... but for parents to first discover that there is a cure...and then to discover that it costs 2.1 milion dolars?!? Horrible... If over 500 kids have been treated in 2020 ... How much more money do you need??? Humanity is lost...

[@MAD4FAK4]

@@adrianamirauta8206 That's exactly what I mean. Some people need to get off their high horses. I understand the time you spent developing the cure, the research, sleepless nights, funding, expenses, stress. But do you understand how many kids will randomly get SMA each year on a global scale? Countless.. If the price for this cure was just the third of this, more kids could live their lives without pain and I guarantee you, you will have more revenue selling more units for less $, rather then pumping up the price and selling a few. It's as simple as that, and whoever was responsible for determining the price needs to study business & management more.

[@wissamsidki5237]

@@MAD4FAK4 some people are so ignorant 😔

[@marojojo507]

I know a child that has SMA and his family is struggling to collect enough money to get the vaccien before he turns tow it so sad I swear they still didn’t have enough.

[@nikilyons8999]

The children in my state get this Gene therapy through the state insurance in Tennessee. At Vanderbilt Bilt Hospital in Nashville

[@mariarzg3786]

What about sma type 2

[@m.m.7518]

Is that zolgensma ((with its very expensive price)) taken once to get healthy children or this is just one dose

[@zanozano9348]

I heard that the frist dose is 125k And then it becomes 325k The overall cost is about 2 million dollars.

[@leenalkhoury5125]

does it work for older people my friend has and she's 26 years old

[@CynicalScientist261]

Treatment can slow progression of SMA in older patients.

[@crybaby3710]

watch squirmy and grubs! they're a wonderful fucking hilarious couple who took the spinal version and now takes the oral version and his muscles got a bit stronger actually ☺ and they're my favorite channel

[@teneeishajones4538]

Hi. I would love to hear more about this. My daughter is 18mths old and we just found out she has SMA. I know there are different types of sma but i would like to know would this treatment work for her.

[@chary361]

You tube Shane Burcaw Squirmy and Grubs

[@creativityhub1350]

If you can't afford it, the genetic sequence that Zolgensma used is public info. Their administration protocol as well.

[@renjunimvlogs7784]

Hw is ur baby now

[@deepakgurung3853]

Hope your baby is fine

[@ruangbersalinrsudsukoharjo8009]

Is it zolgensma??? Very expensive for us..

[@akilshaikh6580]

How much fees

[@creativityhub1350]

2 Million. For people who can't afford it however, the genetic sequence that Zolgensma used is public info. Their administration protocol as well.

[@silsilayeislami1031]

@@creativityhub1350 my son 2 yers age he affected SMA career in banglades plzz help

[@Baronpato]

@@creativityhub1350 how do you mean that this is public info. What can be done with it

[@MAD4FAK4]

@@Baronpato He's just talking out of his ass.. It's like "hey bro it's easy, you just have to release pre-mRNA so that it binds to SMN-2 to prevent exon 7 from getting removed from the chromosome, just show it to your doctor, he'll do it for cheaper". So don't pay attention what he's saying.

[@erdenemashbat3379]

@@MAD4FAK4 I'm guessing it takes a lot to produce the drug?

[@silsilayeislami1031]

my son 2 yers age he affected SMA career in banglades plzz help

[@aditiharris8755]

I hope he is fine now?

[@fatimasalman8921]

​@@aditiharris8755how is your baby now?

[@kimchee94112]

Zolgensma

[@shalabypharmd4137]

I looked it up. It’s 2 million dollars drug. Is that right ?