Explaining the Role of SMN Genes in Spinal Muscular Atrophy

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by Wendy Henderson |

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In this educational video from Roche shared in August 2014, find out more about motor neurons and what happens to the motor neurons in children who have spinal muscular atrophy (SMA).

Scientists measure major development milestones in spinal muscular atrophy. Find out more.

The film talks about the SMN1 and SMN2 genes and how faults in these genes can result in the muscle degeneration associated with SMA. It also briefly touches on some of the mouse model work the organization has been doing to help isolate the SMN2 gene and prevent it from splicing, which may allow for more SMN protein to be produced, leading to the normal development of muscles.

Phase 2 clinical trial of an oral compound targeting the genetic cause of SMA starts in Europe. Read more here.

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