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?

[@MihaiAdrianNegru]

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

[@prembiswas5581]

Pls help me I sma pesent

[@heartless6254]

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

[@pallavmahato7865]

Anyone from NDTV ?

[@shahidbhutto3284]

My sun sma pashant