New treatment offers glimmer of light for spinal muscular atrophy patients: 6 observations

United Kingdom-based University of Oxford researchers developed a new treatment for spinal muscular atrophy, according to Medical Xpress.

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Here are six observations:

 

1. The new treatment, Pip6a-PMO, involves peptide Pip6a delivering the splice-switching oligonucleotide.

 

2. Pip6a has the ability to deliver splice-switching oligonucleotides to many tissues in the body, even into the brain and spinal cords.  

 

3. After injecting SMA-ridden young mice with Pip6a-PMO, they became heavier and grew faster than untreated mice.

 

4. The treated mice lived longer than untreated mice. One dose of 10 microgrammes per gram of weight of Pip6a-PMO allowed the young mice to live for a median 167 days, compared to 12 days for untreated mice.

 

5. Two doses of Pip6a-PMO resulted in mice living at least 200 days with a median survival of 457 days. This reflects a survival period that is 38 times longer than untreated mice.

 

6. The researchers plan to conduct a two-year study involving human patients, which is set to commence in 2017.

 

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