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Cystic fibrosis (CF) is characterized by abnormal ion transport in the apical membrane of epithelial cells. The partnership will use a novel and replication deficient lentiviral vector in

Neuren retains rights to develop and commercialize trofinetide for all indications outside of North America. ACADIA plans to initiate a Phase 3 randomized, double-blind placebo-controlled study evaluating trofinetide

Kapspargo Sprinkle, a U.S. Food and Drug Administration (FDA) approved, novel, patented formulation of metoprolol succinate, is the first and only extended-release sprinkle formulation of this beta1-selective adrenoreceptor

With this decision, eligible adults in the UK with hATTR amyloidosis can gain access to patisiran before the drug is granted marketing authorization by the European Commission (EC).

AstraZeneca licensed selumetinib from Array BioPharma in 2003. AstraZeneca and Merck have entered a co-development and co-commercialisation agreement for selumetinib in 2017. NF1 is an incurable genetic condition,

Under the terms of the agreement, Juvenescence will purchase, in a single transaction, 14.4 million shares of AgeX Therapeutics from BioTime for $43.2 million. 50% of the purchase

The phase 2 trial evaluated ridinilazole against Standard of Care vancomycin across 100 patients. By preserving the gut microbiome, the Summit drug was demonstrated to have brought down

Amgen is evaluating AMG 890 in a Phase 1 clinical study designed to assess its safety in volunteers with elevated levels of lipoprotein (a) (Lp(a)). Emerging research has

The company’s announcement also included the issuance of $22m in shares upon conversion of debt into equity Participants included ARCH Venture Partners, the Wellcome Trust, Steven Paul M.D.,