ATLANTA, Georgia — UCB, a global biopharmaceutical company, today presented positive efficacy and safety results from the GEMZ phase 3 study investigating adjunctive fenfluramine (FFA) in children and adults with CDKL5 deficiency disorder (CDD) at the American Epilepsy Society (AES) meeting in Atlanta, December 5-9, 2025.1 The trial met its primary endpoint and...
treatment News
ATLANTA — UCB (Euronext Brussels: UCB), a global biopharmaceutical company, today announced that it will be presenting results from across its portfolio in generalized myasthenia gravis (gMG) at the American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) annual meeting and the Myasthenia Gravis Association of America Scientific Session taking place...
ATLANTA — UCB, a global biopharmaceutical company, today announced that it will present six abstracts in hidradenitis suppurativa (HS) at the 8th Annual Symposium on Hidradenitis Suppurativa Advances (SHSA) in Phoenix, Arizona. Data and analyses in HS will be presented across two oral presentations and four posters. “The data to...
ATLANTA, Georgia — UCB, a global biopharmaceutical company, today announced that the final analysis of an open-label extension (OLE) study with the objective to describe long-term safety and effectiveness of FINTEPLA (fenfluramine) in Lennox-Gastaut syndrome (LGS) was published in Epilepsy & Behavior.2 No new or unexpected treatment-emergent adverse events (TEAEs) were reported.2 Children and adults...
Chicago, Illinois – The University of Chicago Medicine Comer Children’s Hospital will be among the first in the country to offer gene therapy for sickle cell disease in patients 12 years and older, after federal regulators approved two new treatments in December 2023. Thousands of patients with sickle cell disease...
LOS ANGELES, Calif. – Researchers at UCLA have developed a nonviral gene-editing strategy that could help pave the way for one-time treatments for people with cystic fibrosis (CF). The experimental therapy uses lipid nanoparticles — tiny fatty molecules — to insert a full, healthy copy of the CFTR gene into human airway cells,...
FAIRFAX, Va. — A minimally invasive treatment using MRI and transurethral ultrasound instead of surgery or radiation is effective in treating prostate cancer, according to new research to be presented at the Society of Interventional Radiology Annual Scientific Meeting in Salt Lake City. The traditional treatment options of radiation or...
Los Angeles, Calif. – A study led by UCLA Health Jonsson Comprehensive Cancer Center researchers found that using a combination of experimental immunotherapy drugs with chemotherapy significantly improves progression-free survival and overall survival for patients with metastatic colorectal cancer who have previously undergone standard chemotherapy treatment when compared to those who received the targeted...
San Francisco, Calif. – UCSF Benioff Children’s Hospital Oakland is enrolling patients in an innovative clinical trial that seeks to cure sickle cell disease. The trial is the first in the U.S. to apply non-viral CRISPR-Cas9 gene-editing technology in humans to directly correct the genetic mutation that causes the disease....
PARIS – GenSight Biologics (Paris:SIGHT) (Euronext: SIGHT, ISIN: FR0013183985, PEA-PME eligible), a biopharma company focused on developing and commercializing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders, today announced that its gene therapy LUMEVOQ® has been granted Promising Innovative Medicine (PIM) designation by the UK’s Medicines and...