La ricerca contro la SMA
Results from Isis phase 2 trials

Results from Isis phase 2 trials

Isis Pharmaceuticals released results from their Phase II open-label clinical trials of ISIS-SMNRx in infants and children with spinal muscular atrophy at the 19th International…

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Good news from AveXis

Good news from AveXis

AveXis, a clinic-stage gene therapy company, announced the completion of dosing in the low dose cohort in the world’s first human gene therapy trial for…

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A new trial from Roche

A new trial from Roche

Roche recently updated clinicaltrials.gov with a new trial, “MOONFISH”, investigating the safety and efficacy of an oral SMN2 splicing modifier in SMA patients. This drug,…

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Isis advances into Phase 3 study

Isis advances into Phase 3 study

Isis Pharmaceuticals announced the initiation of a pivotal Phase 3 study evaluating ISIS-SMNRx in SMA infants; the Phase 3 study, ENDEAR, is the first of…

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Top-line results of pivotal trial of olesoxime

Top-line results of pivotal trial of olesoxime

Trophos announced that top-line results from a pivotal clinical trial of its lead product candidate olesoxime in spinal muscular atrophy show a beneficial effect on…

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Drug rush

Drug rush

Now there are five potential drugs against SMA entered clinical development. The SMA drug pipeline continues to update: here's the latest version!  

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LAST UPDATE: November 22, 2014

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This site is dedicated to the memory of Federico Milcovich and all the people who died prematurely due to spinal muscular atrophy and other neuromuscular diseases.
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