La ricerca contro la SMA
Roche launches two new trials

Roche launches two new trials

Roche has officially launched two Phase 2 clinical trials testing RO7034067 (also known as RG7916). The trials will begin recruitment in October. Earlier this month,…

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Towards the approval of nusinersen!

Towards the approval of nusinersen!

Biogen and Ionis Pharmaceuticals provided a statement regarding the decision to submit the first ever SMA regulatory filings for FDA approval. Today marks an important…

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Update on Novartis SMA program

Update on Novartis SMA program

Novartis recently provided the following update on clinical trials for LMI070, an orally available SMA drug that corrects SMN2 splicing: "We have made the difficult…

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Data from Avexis SMA program

Data from Avexis SMA program

AveXis presented an interim analysis of data as of April 1, 2016 from the ongoing Phase 1 trial of AVXS-101 for the treatment of spinal…

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Update on nusinersen

Update on nusinersen

Ionis Pharmaceuticals announced it has completed its target enrollment of the Phase 3 Cherish study, a trial designed to support marketing approval of the drug…

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New therapeutic target for spinal muscular atrophy

New therapeutic target for spinal muscular atrophy

Neuroscientists have discovered a specific enzyme that plays a critical role in spinal muscular atrophy, and that suppressing this enzyme's activity, could markedly reduce the…

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LAST UPDATE: October 1, 2016

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