Crigler Najjar Syndrome, also called hyperbilirubinemia, is a chronic condition characterized by high levels of unconjugated bilirubin in the blood. The disease manifests as Jaundice at birth, and can progressively lead to kernicterus, a condition in which the accumulation of unconjugated bilirubin can damage brain...
LogicBio Therapeutics (NASDAQ: LOGC), a gene-editing company that uses its GeneRide technology platform to develop solutions for rare diseases in pediatric patients was recently awarded an Orphan Drug Designation (ODD) status for its LB-001 a recombinant adeno-associated viral vector with human methylmalonyl-COA mutase (MUT) gene...
Pruritus is characterized by an uncomfortable and persistent urge to scratch caused either due to a skin disorder or an underlying systemic disease such as atopic dermatitis, mycoses, chickenpox, hepatitis, Jaundice, chronic renal failure, cholestasis or NASH. These can be chronic in nature and affect...
