Question

The infantile type (Werdnig-Hoffman) of Spinal Muscular Atrophy has a median age of death of 7 months. Death often occurs within a few years of birth. Life can be prolonged with the antisense drug 'Nusinersen', which modulates the alternative splicing of the SMN2 gene to make it produce as much 'Survival of Motor Neuron' protein as SMN1 gene. What is the median age of death for the infantile type of Spinal Muscular Atrophy?

1) 7 months
2) 1 year
3) 2 years
4) 5 years

Answer 1

The median age of death for infantile type Spinal Muscular Atrophy is normally 7 months, but it can be prolonged with the use of the antisense drug 'Nusinersen'.

Step-by-step explanation:

The median age of death for the infantile type of Spinal Muscular Atrophy (SMA), also known as Werdnig-Hoffman SMA, is 7 months. Usually, death occurs within a few years of birth. However, the antisense drug 'Nusinersen' can prolong life by modulating the alternative splicing of the SMN2 gene, allowing it to produce more 'Survival of Motor Neuron' protein like the SMN1 gene. With this drug, the median age of death can be increased beyond 7 months.