TUCSON, Ariz. — Critical Path Institute (C-Path) is excited to announce the launch of the Critical Path for Alpha-1 Antitrypsin Deficiency (CPA-1) Consortium. The CPA-1 consortium aims to accelerate drug development for alpha-1 antitrypsin deficiency (AATD), a rare disease that affects individuals and families worldwide. This will be achieved by...
research News
Madrid, Spain – Brain metastases are one of the most severe complications of melanoma, the most aggressive type of skin cancer. Researchers at the Institute for Neurosciences (IN), a joint center of the Spanish National Research Council (CSIC) and the Miguel Hernández University (UMH) in Elche, have identified a strategy to...
King of Prussia, Pennsylvania – CSL Behring has released promising 3-year results from the pivotal HOPE-B study, confirming the sustained long-term durability and safety of etranacogene dezaparvovec-drlb (Hemgenix®) in individuals with hemophilia B. The data, demonstrating prolonged elevated factor IX activity levels following a single infusion, were recently presented at...
HATTERSHEIM AM MAIN, Germany – At this year’s fully virtual International Congress of the European Respiratory Society (ERS 2021), CSL Behring will be hosting a highly significant and topical virtual symposium on 6 September 2021, from 20:00 – 21:30 CEST. Focusing on what we have learnt about the impact of global Covid-19 pandemic...
KING OF PRUSSIA, Penn. — Global biotechnology leader CSL today announced the U.S. Food and Drug Administration (FDA) has accepted the company’s Biologics License Application (BLA) for garadacimab (CSL312) as a once-monthly prophylactic treatment for hereditary angioedema (HAE). The company also announced the European Medicines Agency (EMA) has accepted the...
Tsukuba, Japan — Nonfunctional pancreatic neuroendocrine tumors, while rare, are primarily treated through surgery. The presence or absence of lymph node metastasis considerably influences the selection of surgical and other treatment approaches. Particularly controversial is the necessity of surgery for tumors smaller than 2 cm as current clinical guidelines provide...
Longitudinal circulating tumor DNA (ctDNA) analysis can predict disease recurrence and guide adjuvant treatment in patients with resected non-small cell lung cancer (NSCLC), according to a study published in Nature Communications. ctDNA positivity — both after surgery and after adjuvant therapy — was significantly associated with worse recurrence-free survival (RFS), researchers...
NEW YORK, NY — Newly published research in the journal Glia has identified crucial links between dietary choices and the progression of multiple sclerosis (MS). The study, led by Patrizia Casaccia, founding director of the Advanced Science Research Center at the CUNY Graduate Center’s (CUNY ASRC) Neuroscience Initiative and Einstein...
New York, NY – Cure Mito Foundation and Hope for PDCD foundation, both patient-led foundations focused on advancing research and supporting families affected by Leigh syndrome and Pyruvate Dehydrogenase Complex Deficiency (PDCD), respectively, are excited to announce a launch of a PDCD patient registry. This innovative registry will be led...
NEWPORT BEACH, Calif. and EDMONTON, AB — CureDuchenne Ventures announced an initial $1M investment in Entos Pharmaceuticals Inc (Entos), a biotech company that develops genetic medicines utilizing its non-viral, redosable Fusogenix PLV delivery platform. Entos will use this funding to create a muscle-targeting therapeutic to deliver full-length dystrophin for the treatment of Duchenne...