Quantitative transcriptome‐based analysis predicts a combination therapy for severe haemophilia B: A case report.

This article discusses a case report on the treatment of severe haemophilia B using a combination therapy of sodium valproate and sirolimus. The patient, an 18-year-old Chinese male, had been diagnosed with severe haemophilia B at a young age and had experienced joint bleeding and deterioration. Through quantitative transcriptome analysis, the researchers predicted and administered the combination therapy, which resulted in improved motor function and quality of life for the patient. However, the study acknowledges the limitation of a small sample size and the need for further validation and investigation of the mechanisms of action of these drugs. [Extracted from the article]