As a Neurology specialist I have come across so many victims of a bad genetic disease of survival motor neuron gene (SMN 1&2) leading a neuromuscular disorder caused by loss of anterior horn cells. Three subtypes named as Werdnig Hoffman, Dubowitz and Kugelberg Welander disease causes proximal weakness, atrophy, respiratory paralysis and death. A new drug Nusinersen approved by FDA for SMA brings in lots of hope for the victims, except the huge costfactor.
But there is something that we can boast of in ongoing research on degenerative neurological disorders. Share your views on this new drug for a bad disease. Any hopes or is it a false dawn?
