Spinal Muscular Atrophy

Gene therapy is a fitting approach for diseases caused by a single gene mutation, like SMA. It targets the cause of disease by delivering a fully functioning copy of the gene into motor neuron cells. Let’s learn more about how it works.
The subtitling of these videos was funded in part by a donation from Pfizer, Inc.

[@MW2proification]

Since the gene for motor functions mutates for the disease to occur, what causes the mutation? Is it usually such as radiation or mutagens?

[@Beshak123]

its a mutation of the gene SMN1 (survival motor neuron1) as a result the lower motor neurons on the anterior horn die. It is genetic but the onset can still be as an adult rather than as a child (type 4- latest onset and mildest of them)

[@MihaiAdrianNegru]

What can be done for adults that are way in their 20s and still suffer from the SMA?

[@saiyelave4588]

@Otis Rohan And how us this relevant to sma?

[@alfredephraim6773]

i dont mean to be so offtopic but does someone know of a trick to log back into an Instagram account?
I somehow forgot the account password. I would appreciate any tips you can give me.

[@rubengannon1110]

@Alfred Ephraim instablaster :)

[@Beshak123]

it's just symptom management actually, depending on whether breathing/eating is affected

[@pallavmahato7865]

Anyone from NDTV ?

[@heartless6254]

A girl from Nepal is suffering from this one like for that girl
👇

[@hridayabajracharya5047]

You are like your name only begging for like

[@x-tzer6859]

Report gardim

[@dropout4]

She has nothing to do with your like on comment,get a life

[@hridayabajracharya5047]

@@x-tzer6859 gardeu bro

[@saiyelave4588]

no I won't

[@shahidbhutto3284]

My sun sma pashant