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...
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NEW HAVEN, Conn. – Long-term treatment with Cerezyme (imiglucerase), or its discontinued predecessor Ceredase (alglucerase), led to rapid and progressive improvements in blood counts, growth, and bone health, as well as reductions in liver and spleen size, in children with Gaucher disease (GD) — including among youngsters with a more severe disease type....
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...
INDIANAPOLIS — A study of more than 21,000 average risk patients at 186 sites across the U.S., led by Regenstrief Institute and Indiana University School of Medicine research scientist Thomas Imperiale, M.D., has found that the next generation multi-target stool DNA colorectal cancer screening test detects 94 percent of colorectal...
SAN DIEGO, Calif. — Regulus Therapeutics Inc. (Nasdaq: RGLS), a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs, today announced it has completed enrollment in the third cohort of patients in the Phase 1b MAD study of RGLS8429 for the treatment of ADPKD. “The completion of our...