Cydan Development Inc. (Cambridge, Mass.), which operates an accelerator that spins out rare genetic disease companies, raised $34 million on Oct. 17 in a round led by existing investor New Enterprise Associates (NEA). New investor...

Cydan Development Inc. (Cambridge, Mass.), which operates an accelerator that spins out rare genetic disease companies, raised $34 million in a round led by existing investor New Enterprise Associates (NEA). New investor Longitude Capital and...

Vtesse Inc. is hoping to change the course of disease in Niemann-Pick type C, a fatal ultra-Orphan lysosomal storage disorder, using a compound plucked from NIH's Therapeutics for Rare and Neglected Diseases repurposing project. Niemann-Pick...

FDA granted Orphan Drug designation to the hospital for Trappsol hydroxypropyl-beta-cyclodextrin (HPBCD) to treat Niemann-Pick type C disease. Children's Hospital & Research Center , Oakland, Calif. Product: Trappsol hydroxypropyl-beta-cyclodextrin ( HPBCD ) Business: Metabolic WIR Staff...

Data from an open-label Phase I trial evaluating a 37.5 mg IV bolus of Dyloject in 13 patients with renal impairment showed that 8 patients with hepatic impairment and 19 healthy volunteers showed no significant...