October 10, 2016.
Researchers at the University of London are currently developing a new therapy to treat spinal muscular atrophy: Nusinersen. A ray of hope for people affected by this disease.
Testing an investigational drug
Spinal muscular atrophy (SMA) is a rare genetic disease, which is characterized by atrophy of the muscles. It can vary from patient to patient and is more or less severe, but it often leads its carriers to early death.
In order to treat young patients, a new investigational drug, Nusinersen, has been tested. He had very good results and even made it possible for several patients to walk again.
Hope for other diseases
It is Professor Francesco Muntoni of the University of London and his colleagues who are working on this new treatment, an antisense oligonucleide. In this therapy, a fragment of DNA is used to target certain RNAs and modify the proteins produced.
Although it is not yet proven that the Nusinersen is the answer to spinal muscle atrophystudies have shown that after being injected into mice, the drug spread throughout the brain and spinal cord. If these clinical results prove to be conclusive, antisense treatments like Nusinersen could bring hope for patients with Alzheimer’s disease or Huntington’s disease.
