La ricerca contro la SMA
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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Interim results from Isis phase 2 trials

Interim results from Isis phase 2 trials

Isis Pharmaceuticals provided an update on its ongoing open label, multiple dose Phase 2 study of ISIS-SMNRx in infants with spinal muscular atrophy. To date,…

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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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Roche and PTC enter clinical stage

Roche and PTC enter clinical stage

PTC Therapeutics, the SMA Foundation and Roche announced today that their joint research program in spinal muscular atrophy has entered the first stage of clinical…

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Novel target for potential SMA therapy

Novel target for potential SMA therapy

A recent study led by scientists from the National University of Singapore (NUS) opens a possible new route for treatment of spinal muscular atrophy.Using global…

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Gene therapy

Gene therapy

Gene therapy is an approach to treating diseases by either replacing faulty genes, modifying the expression of an individual's normal genes, or correcting abnormal genes.…

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Disclaimer


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: March 11, 2014

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CAUTION Medical contents on the site are purely for guidance and information and cannot replace in any case the medical advice. All contents provided by the site are written exclusively by professionals in the medical-scientific area, unless an explicit statement does not specify otherwise.
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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.