Targeted treatment shows promise for severe spinal muscular atrophy
Antisense drug nusinersen was well tolerated in trial of 20 babies with SMA, with majority showing improved muscle skills and function after treatment.
Source: Garo / Phanie / Science Photo Library
Infantile-onset spinal muscular atrophy (SMA) is the most severe form of the disease, and most babies diagnosed with the condition don’t live beyond two years without breathing and feeding support.
Nusinersen is an antisense drug that redirects a ‘back-up’ gene (SMN2) to produce a protein that is deficient in babies with SMA.
In a phase II open-label trial, researchers explored the effect of nusinersen delivered by lumbar puncture to 20 babies with SMA aged between three weeks and seven months.
The authors also observed improvement in muscle skills and function in the majority of babies and 16 babies survived without needing permanent breathing support.
The researchers conclude that their findings provide proof of principle for the use of antisense drugs such as nusinersen in neurological disorders.
Citation: Clinical Pharmacist DOI: 10.1211/CP.2017.20202122
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