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Avoiding intubation and mechanical ventilation during subcutaneous pump implantation under local anesthesia and conscious analgosedation was feasible in patients with advanced pulmonary arterial hypertension (PAH), according to the results of a recent study published in Journal of Infectious Diseases. Researchers retrospectively reviewed outcomes of patients with PAH undergoing a surgical procedure...
St. Jude Children’s Research Hospital scientists looking for drugs to improve survival of children with high-risk neuroblastoma found a promising candidate in CX-5461. Then researchers identified safety concerns with the experimental drug that have implications for current clinical trials in adults. The study appears today in Nature Communications. CX-5461 is...
CAMBRIDGE, Mass.– Sage Therapeutics, Inc. (Nasdaq: SAGE), a biopharmaceutical company leading the way to create a world with better brain health, today announced the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation (ODD) to SAGE-718 for the treatment of Huntington’s disease (HD). SAGE-718 is in development as a...
SHANGHAI, China — Gannex Pharma Company Ltd, a wholly-owned company of Ascletis Pharma Inc., announced that its strategic partner, Sagimet Biosciences Inc., today reported positive topline results from Sagimet’s FASCINATE-2 Phase 2b clinical trial of denifanstat versus placebo in biopsy-confirmed non-alcoholic steatohepatitis (NASH) patients with stage 2 or stage 3...
LEXINGTON, Mass. — SalioGen Therapeutics, a biotechnology company developing next-generation genetic medicines based on its novel Gene Coding™ technology, today announced the nomination of a development candidate, SGT-1001, for the treatment of Stargardt disease, a rare and progressive inherited retinal disease. SGT-1001 is being developed as a one-time, non-viral therapy to...
Jurassic Park actor Sam Neill was recently interviewed by The Guardianopens  in advance of the release of his memoir, Did I Ever Tell You? Originally planned as a glimpse into his career, his life as a celebrity, and his farm in New Zealand, the book takes a dark turn in...
PENNSVILLE TWP. — Sandy McWilliams, a former Pennsville resident, will walk 700 miles this summer from her home in Kentucky to her mother’s house in Pennsville to raise awareness on progressive supranuclear palsy, a rare disease that her mother is fighting. PSP is a rare, fatal brain disease that McWilliams’...
BRISBANE, Calif. – Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, today announced that the European Medicines Agency’s Committee for Orphan Medicinal Products (COMP) released details supporting the Orphan Designation of BIVV003, an investigational ex vivo gene-edited cell therapy product candidate currently being evaluated for the treatment of sickle...