Albireo Pharma, Inc. (NASDAQ: ALBO) a clinical-stage company targeting rare liver diseases, announced the acceptance of its New Drug Application (NDA) for odevixibat, a once-daily, non-systemic IBAT, intended for the treatment of pruritus in patients with progressive familial intrahepatic cholestasis (PFIC). The candidate is also...
Shares of BioCryst Pharmaceuticals, Inc. (NASDAQ: BCRX) are rallying after a discussion in r/wallstreetbets called it the “most undervalued stock in the market.” In January 2021, the Company had reported the grant of a marketing and manufacturing approval for oral, once-daily ORLADEYO™ (berotralstat) 150 mg for...
Axovant Gene Therapies (NASDAQ: AXGT) announced the grant of an Orphan Drug Designation for AXO-AAV-GM1, the Company’s investigational gene therapy for the treatment of GM1 gangliosidosis. AXO-AAV-GM1 is capable of restoring the β-galactosidase enzyme activity by delivering a functional copy of the dysfunctional GLB1 gene,...
Ovid Therapeutics (NASDAQ: OVID), a biopharmaceutical company developing novel therapeutics for rare neurological disorders, announced the launch of a crucial phase 3 clinical trial in Angelman Syndrome named NEPTUNE. OV101, its lead candidate for the treatment of syndrome and Fragile X Syndrome is a delta-selective...
Severe combined immunodeficiency (SCID) or “Bubble Boy Syndrome” is a rare genetic disorder caused by gene mutations that inhibit the functioning of immune cells. Children afflicted with this condition are healthy in appearance but are susceptible to severe infections, even a common cold can prove...
