UK osteopetrosis drug granted FDA Orphan Drug status
RNA delivery and therapeutics company SiSaf Ltd has announced that its siRNA therapeutic for patients with Autosomal Dominant Osteopetrosis Type 2 (ADO2) has been granted Orphan Drug Designation by the FDA. In addition, due to the serious manifestations of this rare skeletal disorder in children, SIS-101-ADO has been granted Rare Pediatric Disease Designation for the […]