SMA Newsroom

EMA validation of the Marketing Authorisation Application

Scholar Rock: Expanded Access Program

<p>Scholar Rock announced that the FDA has granted priority review for the Biologics License Application for apitegromab, an investigational muscle-targeted treatment developed to improve motor function for people living with SMA who are receiving an SMN-targeted treatment.</p>

Community update from Scholar Rock

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<p>Novartis shared updates on the results from Phase III STEER and Phase IIIb STRENGTH studies, evaluating intrathecal onasemnogene abeparvovec (OAV101 IT) in people living with SMA aged two to &lt;18 years.&nbsp;</p>

Community update from Novartis: Phase III STEER and Phase IIIb STRENGTH studies

<p>Roche shared updates on the final results of the now-concluded SUNFISH study, which assessed the efficacy and safety of risdiplam in people living with SMA aged 2-25 years.</p>

Community update from Roche: Final five-year data from the pivotal SUNFISH (NCT02908685) study 

Call for Research Lay Summary of the Projects

Call for Research website

Our Call for Research Projects

<h2><strong>We are proud to announce the five scientific projects that have been selected for funding through our Call for Research no. 12.</strong></h2>
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SMA Newsroom

Community update from Scholar Rock

Community update from Novartis: Phase III STEER and Phase IIIb STRENGTH studies

Community update from Roche: Final five-year data from the pivotal SUNFISH (NCT02908685) study

Call for Research no. 12. Learn about the shortlisted projects!

We are proud to announce the five scientific projects that have been selected for funding through our Call for Research no. 12.

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