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New therapeutic development showing promise for infants with SMA

gettyimages-86802223The early cancellation of the clinical trial for the experimental drug nusinersen found that the difference in the children given the drug, and those on the placebo was so drastic that doctors determined they could no longer administer the placebo in good conscience. The drug nusinersen is designed to help those affected by spinal muscular atrophy (SMA).

Dr. Craig Campbell is a scientist/paediatrician with the Children’s Hospital at London Health Sciences Centre who works closely with patients affected by SMA, and has seen first hand the encouraging signs the treatment has given some of his patients. We asked Dr. Campbell a few questions regarding the news of this clinical trial:

What is nusinersen and how does it work?

Nusinersen is an antisense oligonucleotide, which is basically a small piece of genetic material that works by tricking the cell machinery into reading and producing a normal SMN protein, from another gene that is very similar to the one missing in patients with SMA.

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