Decreased levels of an enzyme lead to cellular dysfunction and neuronal degeneration involved in the development of spinal muscular atrophy (SMA), a new mouse study suggests. The study, “Neuronal activity regulates DROSHA ... Read more
Roche has stopped the development of olesoxime, a treatment candidate for spinal muscular atrophy (SMA) following disappointing 18-month results in an ongoing clinical study, the company announced. In a statement ... Read more
Scholar Rock announced it has completed dosing a first group of healthy volunteers in its Phase 1 clinical study of SRK-015, a spinal muscular atrophy (SMA) treatment candidate. SRK-105 selectively blocks the activation ... Read more
A potential oral treatment for children with type 1 spinal muscular atrophy (SMA) called RG7916 was able to safely raise levels of the crucial SMN protein that is lacking in these patients, ... Read more
Ionis Pharmaceuticals has received $40 million in milestone earnings from Biogen following Japanese regulatory approval of the price of Spinraza, Biogen’s treatment for spinal muscular atrophy.
So far, Ionis has earned more ... Read more
Spinal muscular atrophy (SMA) is a genetic neuromuscular disease. SMA patients have either a missing or altered gene, (the survival motor neuron-1 or SMN1 gene), which causes large motor nerves ... Read more
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