Jordan Janz used to take 40 to 60 pills every day to treat a rare genetic disease. But a side effect of the medicine made the 22-year-old smell bad. Janz is now taking an experimental gene treatment. He thought the treatment might be worth it when he went to work and his...
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BOSTON, Mass. – A novel gene therapy designed to target a form of inherited deafness restored hearing function in five children who were treated in both ears. The children also experienced better speech perception and gained the ability to localize and determine the position of sound. The study, the world’s...
Cambridge, Mass. – Cystic fibrosis is one of the most common genetic disorders, causing thick mucus build-up in the lungs and other parts of the body, breathing problems, and infection. A three-drug cocktail known as Trikafta has greatly improved patient quality of life since its development in 2019, but can...
An international, multidisciplinary team of researchers from the Translational Synthetic Biology Laboratory at Pompeu Fabra University (Barcelona, Spain), led by Dr. Marc Güell, has published an article in the scientific journal Nature Communications showing the potential of Find Cut-and-Transfer (FiCAT) technology as a state-of-the-art tool for gene writing to develop advanced therapies that are safer and more...
Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced new interim data from two studies of Evrysdi® (risdiplam); JEWELFISH and RAINBOWFISH. Data from JEWELFISH, an ongoing open-label study primarily evaluating the safety of Evrysdi in people aged 1 to 60 years who have been previously treated...
Memphis, TN – Scientists from St. Jude Children’s Research Hospital, Seattle Children’s and the Children’s Oncology Group (COG) have identified novel genetic variations that influence relapse risk in children with standard risk B-cell acute lymphoblastic leukemia (SR B-ALL), the most common childhood cancer. The identification of genomic predictors of relapse...
COPENHAGEN, Denmark – Genmab A/S (Nasdaq: GMAB) today announced new and updated data from three arms of the ongoing Phase 1b/2 EPCORE® CLL-1 trial (NCT04623541) evaluating the efficacy and safety of epcoritamab-bysp, a T-cell engaging bispecific antibody administered subcutaneously, as a monotherapy and in combination for the treatment of patients with Richter transformation (RT), a rare complication in which chronic lymphocytic leukemia (CLL) evolves into an aggressive lymphoma, most often diffuse...
BERLIN, Germany – Lambert-Eaton myasthenic syndrome (LEMS) can be caused by several types of cancer, not just the small-cell lung cancer (SCLC) that’s commonly associated with the immune system disease, according to new data from a European LEMS registry. These data showed that, compared with autoimmune LEMS, which occurs on its...
CONCORD, Mass. — ITF Therapeutics LLC, the U.S. affiliate of Italfarmaco, today announced that results on givinostat for the treatment of Duchenne muscular dystrophy (DMD) are being presented at the 2025 Neuromuscular Study Group (NMSG) Annual Scientific Meeting held September 26-28 in Stresa, Italy. Poster presentations include analyses of functional outcomes based on weight-based flexible...
SAN FRANCISCO, Calif. — The question of what causes complex neurological diseases such as Alzheimer’s or multiple sclerosis continues to confound scientists and doctors, with the unknowns standing in the way of early diagnoses and effective treatments. Even among identical twins who share the same genetic risk factors, one may...