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.

[ruthdanielson]

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

[jscarter]

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!!

[faisalmasood]

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

[Kevo]

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.

[teachrare]

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

[TFN_I]

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

[nishanakarmi]

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

[kattarhindu]

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

[teneeishajones]

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.

[m.m.]

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

[leenalkhoury]

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

[ruangbersalinrsudsukoharjo]

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

[dkkazioffice]

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