BioSpace News Archive

ltragenyx Pharmaceutical Inc.(NASDAQ:RARE) today announced that the Committee for Medicinal Products for Human Use(CHMP), the scientific committee of the European Medicines Agency (EMA), has adopted a positive opinion recommending the marketing authorization under exceptional circumstances of Mepsevii™ (vestronidase alfa) for the treatment of non-neurological manifestations of Mucopolysaccharidosis VII (MPS VII; Sly syndrome).

Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), Kyowa Hakko Kirin Co. Ltd, and Kyowa Kirin International PLC today announced that the results of a pediatric Phase 2 clinical trial of Crysvita® (burosumab) for the treatment of X-linked hypophosphatemia (XLH) in children aged 5 to 12 years were published online by the New England Journal of Medicine (NEJM).

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the U.S. Food and Drug Administration (FDA) approved KALYDECO® (ivacaftor) to include use in children with cystic fibrosis (CF) ages 12 to <24 months who have at least one mutation in their cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to KALYDECO based on clinical and/or in vitro assay data.

ltragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, Kyowa Hakko Kirin Co. Ltd(Kyowa Hakko Kirin), and Kyowa Kirin International PLC (Kyowa Kirin International) today announced that the U.S. Food and Drug Administration (FDA) has approved Crysvita® (burosumab-twza) for the treatment of X-linked hypophosphatemia (XLH) in adult and pediatric patients one year of age and older.

Cresco Labs announced today that CEO Charlie Bachtell will be speaking at the Benzinga Cannabis Capital Conference being held in Toronto this week at the Arcadian. He will lead a discussion on how to execute unique cannabis business ideas in the fast-emerging industry.

Neurocrine Biosciences, Inc.(NASDAQ: NBIX) today announced it will present data from its movement disorders programs, including additional long-term results for INGREZZA® (valbenazine) capsules, the first U.S. Food and Drug Administration (FDA) approved treatment for adults with tardive dyskinesia (TD), and data analyses from a Phase III study on opicapone, an investigational treatment for Parkinson’s disease.

Kewaunee Scientific Corporation announced that the Company plans to release its first quarter fiscal year 2019 financial results on Tuesday, August 28, 2018 after the close of trading.

Marathon Kids announced the official opening of their Marathon Kids national grant for the 2018-2019 school year.

Regional Health Properties, Inc. filed its Quarterly Report on Form 10-Q with the Securities and Exchange Commission for the three month period ended June 30, 2018.

DNAe, the inventor of semiconductor-based genomic analysis technologies, and the developer of a new game-changing test for bloodstream infections (BSI) that can lead to sepsis, today announced that it has been shortlisted as a finalist for the 2018 OBN Awards, in the category ‘Best Diagnostic Medtech Company’.