Following this puts new work involving it at the top of your briefing, with a note saying why it is there. Links are taken from the source record, never inferred.
Non-interventional pilot study in 20 patients without a control arm, reporting motor and pain improvements with pridinol added to botulinum toxin, but lacking comparative evidence and power to support clinical decision-making.
Model-based cost-effectiveness analysis demonstrates mavacamten meets accepted Dutch societal willingness-to-pay thresholds using established Markov modeling methods and published trial data.