La ricerca contro la SMA
Update on Novartis SMA program

Update on Novartis SMA program

Novartis has released the following update for the LMI070 clinical trial, on behalf of the program team. Dear SMA community, for years, our researchers have…

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Update on trial Sunfish

Update on trial Sunfish

Genentech/Roche recently provided the following community statement with clinical trial updates for SUNFISH (Type 2/3). The SUNFISH trial is advancing to part two, which is…

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Spinraza: approved!

Spinraza: approved!

The European Commission has granted a marketing authorization for SPINRAZA® (nusinersen) for the treatment of 5q spinal muscular atrophy, the most common form of the…

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New compound can help SMA motor-neurons

New compound can help SMA motor-neurons

Researchers at the Harvard Stem Cell Institute have identified a compound that can restore the SMN motor-neuron protein whose shortage is associated with spinal muscular…

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AveXis announces new pivotal study

AveXis announces new pivotal study

AveXis announced that a new UE pivotal study with AVXS-101 in spinal muscular atrophy Type 1 patients will reflect a single-arm design, using natural history…

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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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This site is aimed at everyone involved in the fight against spinal muscular atrophy, whether patients and their families, physicians, health professionals or students of the area. The information in this site serves to enhance, not replace, the doctor-patient relationship.
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LAST UPDATE: November 29, 2017

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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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