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SHANGHAI, China — VISEN Pharmaceuticals (VISEN), an innovative biopharmaceutical company focused on endocrine diseases, today announced that the Biologics License Application (BLA) for Lonapegsomatropin (TransCon hGH) was accepted by the China National Medical Products Administration (NMPA). Lonapegsomatropin is the first once-weekly administrated growth hormone approved by both the U.S. Food...
In a touching turn of events, the Vodafone Ghana Foundation has made a profound impact on the life of Oleki Gaduga, a 12-year-old girl from Ada. Oleki was born with Infantile Blount Disease, a rare disorder that causes leg bowing and difficulty walking. Oleki had been suffering from severe pain...
Urbana-Champaign, Illinois – The Speech Accessibility Project is now recruiting U.S. and Puerto Rican adults with cerebral palsy. Those interested can sign up online. Funded by Big Tech companies Amazon, Apple, Google, Meta, and Microsoft, the University of Illinois Urbana-Champaign aims to train voice recognition technologies to understand people with diverse...
CAMBRIDGE, Mass. – Vor Biopharma (Nasdaq: VOR or the Company), a cell therapy company pioneering engineered hematopoietic stem cell (eHSC) therapies combined with targeted therapies for the treatment of cancer, today announced the formation of a collaboration with Janssen Biotech, Inc. (“Janssen”), one of the Janssen Pharmaceutical Companies of Johnson...
CAMBRIDGE, Mass. – Voyager Therapeutics, Inc. (Nasdaq: VYGR), a clinical-stage gene therapy company developing life-changing treatments for severe neurological diseases, today announced the U.S. Food and Drug Administration (FDA) has removed its clinical hold on the company’s Investigational New Drug (IND) application for VY-HTT01, a gene therapy candidate for the...
LEXINGTON, Mass. — Voyager Therapeutics, Inc. (Nasdaq: VYGR), a biotechnology company dedicated to advancing neurogenetic medicines, today announced new data from its two preclinical programs targeting pathological tau for the treatment of Alzheimer’s disease. Data on VY-TAU01, Voyager’s lead anti-tau antibody candidate, and on Voyager’s tau silencing gene therapy program...
Cambridge, England – Voydeya (danicopan) has been approved in the US as add-on therapy to ravulizumab or eculizumab for the treatment of extravascular haemolysis (EVH) in adults with paroxysmal nocturnal hemoglobinuria (PNH). Voydeya is a first-in-class, oral, Factor D inhibitor developed as an add-on to standard-of-care Ultomiris (ravulizumab) or Soliris...
WILMINGTON, Del. —  AstraZeneca Pharmaceuticals LP and Ionis’ WAINUA™ (eplontersen) has been approved in the US for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults, commonly referred to as hATTR-PN or ATTRv-PN. WAINUA is the only approved medicine for the treatment of ATTRv-PN that can be self-administered...