We were unable to process your request. Please try again later. If you continue to have this issue please contact [email protected]. Back to Healio Topline results from a phase 3 clinical ...

3D rendered Illustration of non-striated Myocytes, non-skeletal, smooth Muscle cells building muscle tissue. Apitegromab is a selective inhibitor of the inactive form of myostatin to block its ...

"Our preliminary findings show that German children with SMA, despite significant physical disability, have surprisingly good subjective HRQoL." – Erik Landfeldt, MSc, PhD Previous studies have found ...

- Motor function improvements were observed for all three SRK-015 treatment cohorts in the primary efficacy endpoints (Hammersmith scale scores) at six-months; top-line data from the 12-month ...

In Part 2 of the trial, patients 2-25 years of age received either risdiplam for 24 months or placebo for 12 months followed by risdiplam for 12 months. Treatment with risdiplam was associated with ...

Scholar Rock Holding Corp.'s stock soared more than 315% Monday to put it on track for its biggest ever one-day gain, after the clinical-stage biotech company said a late-stage trial of a treatment ...

– Long-term efficacy data from the pivotal SUNFISH study confirm increases in motor function are sustained at three years while adverse events decreased over the same period – – Part 2 of SUNFISH ...

Elaine Chen covers biotech, co-writes The Readout newsletter, and co-hosts STAT’s weekly biotech podcast, The Readout Loud. You can reach Elaine on Signal at elaineywchen.70. The biotech company ...