undisclosed pediatric rare disease therapeutic (rare disease/kidney disease)

undisclosed pediatric rare disease therapeutic (rare disease/immune disease)

undisclosed pediatric rare disease program (rare CNS diseases)

undisclosed pediatric rare disease program (cardiovascular disease)

Opaganib

C₂₃H₂₅ClN₂O

pediatric disease therapeutic

undisclosed rare disease therapeutic (rare disease/eye disease/skin disease)

Rare Disease Therapy (Metis Pharmaceuticals)

LUNAR-RARE2

trimethylangelicin (Rare Partners)