We were unable to process your request. Please try again later. If you continue to have this issue please contact customerservice@slackinc.com. Topline results from a phase 3 clinical trial showed an ...

SMA type 2, previously known as Dubowitz disease, is a hereditary disease that affects motor neurons. It typically becomes noticeable around the ages of 6 to 18 months, when children with the ...

The least-squares mean difference in the Hammersmith Functional Motor Scale-Expanded change from baseline at 12 months was 1.8 points for those 2 to 12 years receiving apitegromab vs placebo.

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Spinal muscular atrophy with respiratory distress (SMARD) is a rare genetic condition that typically affects infants and children. It causes muscle weakness and breathing problems. Spinal muscular ...

Researchers evaluated oxygen saturation and arterial CO2 variables to screen for sleep disordered breathing in children with SMA type 1-3. Of the measures evaluated, only an oxygen desaturation index ...

A positive effect of nusinersen treatment on motor function in ambulant pediatric and adult spinal muscular atrophy (SMA) patients during a 38-month period has been demonstrated by an analysis of data ...

Scholar Rock Holding Corporation (NASDAQ:SRRK) President & CEO Jay Backstrom presented at the J.P. Morgan Healthcare Conference. The discussions mainly focused on apitegromab for spinal muscular ...

A new qualitative participatory study explored how powered mobility (PM), in the form of modified ride-on cars, impacts children with spinal muscular atrophy type 1 (SMA1) and their families. Here are ...

India makes many of the world’s drugs, but treatments for rare diseases like spinal muscular atrophy are imported and prohibitively costly. In desperation, parents are raising funds on social media.