Dublin, Ireland. Boosting levels of a deficient protein has clear survival benefits for people with the genetic condition alpha-1 antitrypsin deficiency (AATD), according to new research led by RCSI University of Medicine and Health Sciences. The findings underscore a call to make augmentation therapy accessible to patients with AATD in...
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Dublin, Ireland – A new partnership between RCSI University of Medicine and Health Sciences and Irish diagnostics company Serosep Ltd is set to personalise treatment for ulcerative colitis and ultimately improve quality of life for patients with the condition. Under the new alliance, RCSI researchers will work with Serosep to...
LA JOLLA, CA — Nerve cells in the brain demand an enormous amount of energy to survive and maintain their connections for communicating with other nerve cells. In Alzheimer’s disease, the ability to make energy is compromised, and the connections between nerve cells (called synapses) eventually come apart and wither,...
Amsterdam, Netherlands – Findings from a new study in the Journal of Neuromuscular Diseases, published by IOS Press, demonstrate the effectiveness of disease-modifying treatments (DMTs) in infants with spinal muscular atrophy (SMA). The study’s results add further support for gene therapy as a treatment modality that can deliver durable transformative...
BRIDGEWATER, N.J. — Recordati Rare Diseases Inc. today announced the presentation of new data detailing advances in the understanding of Castleman disease (CD), a group of rare immune system disorders characterized by abnormal lymph node enlargement and systemic inflammatory symptoms that can be potentially life-threatening. In a series of poster presentations...
SALT LAKE CITY, Utah — Recursion, a leading clinical stage TechBio company decoding biology to industrialize drug discovery, today announced it has signed an agreement with Bayer AG to in-license a new chemical entity that emerged from the companies’ fibrosis research collaboration. The compound represents a novel approach to treating...
TEL-AVIV, Israel and RALEIGH, N.C. — RedHill Biopharma Ltd. a specialty biopharmaceutical company, today announced that its two novel, oral host-directed investigational drugs, opaganib and RHB-107 (upamostat) demonstrated robust synergistic effect when combined individually with remdesivir (Veklury), significantly improving viral inhibition while maintaining cell viability, in a new U.S. Army-funded...
WASHINGTON, DC – Patients with chronic lymphocytic leukemia (CLL) and non-Hodgkin lymphomas (NHLs) who received frequent tests for immunoglobulin G (IgG) were less likely to experience severe infections compared with those who didn’t, according to a study published in Blood Advances. Moreover, only half of patients undergo such testing. Patients...
Guelph, Canada – A new editorial paper was published in Oncotarget entitled, “Reductive carboxylation of glutamine as a potential target in acute myeloid leukemia.” In this new editorial, researchers Alessia Roma, Lawrence D. Goodridge and Paul A. Spagnuolo from the University of Guelph discuss acute myeloid leukemia (AML) — an...
Tarrytown, NY – Along with resolving site inspection issues that cost Regeneron a high-dose Eylea launch in June, the company has scored an FDA nod for Veopoz as the first treatment for the ultra-rare inherited immune condition Chaple disease. The disease, which is also known as CD55-deficient protein-losing enteropathy, has a global patient...