La ricerca contro la SMA
A small molecule for Novartis

A small molecule for Novartis

Researchers at the Novartis Institutes for BioMedical Research (NIBR) report a potential solution of the underlying genetic problem of SMA patients; it was published online…

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Update on trial Moonfish

Update on trial Moonfish

Roche, PTC and the SMA Foundation recently provided an update on the clinical development of RG7800, an investigational SMN2 splicing modifier that is being studied…

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Subcutaneous ASO drug administration

Subcutaneous ASO drug administration

Cold Spring Harbor Laboratory researchers have discovered that a drug to treat SMA in mouse models is effective even when injected subcutaneously, and it is…

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Pfizer terminated its partnership deal with Repligen

Pfizer terminated its partnership deal with Repligen

Pfizer announced its decision to terminate the collaboration deal with Repligen Corporation for developing treatments for spinal muscular atrophy, returning some early stage treatments to…

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Roche buys France's Trophos to expand in neuromuscular disease

Roche buys France's Trophos to expand in neuromuscular disease

Swiss drugmaker Roche has agreed to buy privately-held Trophos to gain access to experimental drug olesoxime to treat spinal muscular atrophy. The acquisition is the…

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LAST UPDATE: September 1, 2015

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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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