According to scientists, new research on ALS may shed light on the mechanisms underlying spinal muscular atrophy.
Two RNA binding proteins, TDP-43 and hnRNP A1, are abnormal in certain cases ... Read more
Treatment with Spinraza (nusinersen) induced significant and clinically relevant motor function improvement in children with later-onset spinal muscular atrophy (SMA), final results of a Phase 3 trial show. The ... Read more
Although Spinraza has the potential to change the course of spinal muscular atrophy, a number of ethical challenges revolve around its use, a group of American researchers argues.
In an article ... 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) patients in the European Union will soon have access to Spinraza (nusinersen), the first treatment approved for people with this disease and one able to treat ... Read more
https://www.youtube.com/watch?v=bVi9L_3LWw4 In this heartwarming video from Robert Harding, we meet Cameron. Cameron was diagnosed with type 1 spinal muscular atrophy (SMA) at five weeks old and was given just ... Read more
In a late-breaking session at the 2016 World Muscle Society Congress in Granada, Spain, Biogen and Ionis Pharmaceuticals presented new data from clinical trials for their co-developed drug candidate ... Read more
Biogen and Ionis Pharmaceuticals recently reported that nusinersen, an antisense drug designed to treat spinal muscular atrophy (SMA), ably met the primary endpoint set for an interim analysis of its Phase 3 ENDEAR clinical ... Read more
Biogen and Ionis Pharmaceuticals have recently issued several updates on the clinical development of nusinersen, an antisense drug designed to treat spinal muscular atrophy (SMA). The partners announced that the ... Read more
Ionis Pharmaceuticals recently presented an update of its ongoing open-label Phase 2 clinical study of nusinersen, often called Ionis-SMN, in infants with spinal muscular atrophy (SMA) at the 2016 American Academy of Neurology ... Read more
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