Happy Monday!
This week, we have stories on a potential treatment approach for an aggressive form of mitochondrial disease, an experimental treatment for osteosarcoma earning Rare Pediatric Disease designation, and a baby born with a rare disease called…CHAOS?
Study of the Week: Scientists Discover a Potential Therapy for an Aggressive Form of Mitochondrial Disease
Scientists may have discovered a method for treating rare and aggressive FBXL4 mitochondrial disease.
Dovitinib for Osteosarcoma Earns Rare Pediatric Disease Designation
The experimental treatment dovitinib, which is being developed for osteosarcoma, has earned Rare Pediatric Disease designation from the US Food and Drug Administration (FDA).
After Four Miscarriages, Finally a Heartbeat – Then – CHAOS
This was the fifth time Yadi Martin was pregnant. The first four pregnancies ended in miscarriages. But this time it was different. The fetus had a heartbeat. That was until a routine anatomy scan alerted the Martin’s doctor that their baby has a rare disease called congenital high airway obstruction syndrome (CHAOS).
