Spotlight Innovation announced that the nonprofit Cure SMA has awarded $300,000 to Prof. Kevin Hodgetts, of Brigham and Women’s Hospital in Boston and a Spotlight research collaborator, for the ... Read more
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
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