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Published on October 7, 2024
After a 30-year wait, the last decade has seen a number of therapies for treating Duchenne muscular dystrophy (DMD) get FDA approval, either by the normal or accelerated pathway, with many more in development. DMD is a recessive, degenerative muscle disorder that almost exclusively affects males or people with one…
Published on June 28, 2024
AbbVie this week acquired Celsius Therapeutics, whose focus is on “pioneering new precision medicines in inflammatory disease by harnessing the power of single-cell RNA sequencing and human biology at scale.” AbbVie acquired all outstanding Celsius equity for $250 million in cash. “AbbVie shares our excitement about the potential of TREM1…
Published on June 30, 2023
The first gene therapy for hemophilia A has finally reached the U.S. This week the FDA approved BioMarin’s Roctavian (valoctocogene roxaparvovec), an adeno-associated virus (AAV) vector-based gene therapy for the treatment of adults with severe hemophilia. The treatment reduced means annualized bleeding rate from 5.4 bleeds per year at baseline…
Published on June 28, 2023
Research led by investigators at Cincinnati Children’s Hospital Medical Center and the University of Cincinnati School of Medicine has revealed new, specific biomarkers of kidney transplant rejection. These specific cellular signals, revealed by single-cell analysis represent new therapeutic and diagnostic targets to improve care for the roughly 10% of patients…
Published on June 14, 2023
Researchers at the Cold Spring Harbor Laboratory (CSHL), have discovered that apart from shrinking pancreatic and colorectal tumors, keto-dieting also accelerates a lethal wasting condition called cachexia in mice models with cancer. However, pairing a steroid treatment with the diet may solve this problem. The keto diet is one of…
Published on June 14, 2023
Results from a study led by researchers at the Garvan Institute of Medical Research in Australia show how treatment with anti-inflammatory steroids can affect a gene called RELA and trigger diabetes symptoms. The results of the research, published in the journal Diabetologia, suggest that RELA plays a joint role in…
Published on June 5, 2023
Chimeric antigen receptor T cell therapy, commonly referred to as CAR T therapy, is an intervention that uses a patient’s own cells to fight their cancer. The therapy first received US Food and Drug Administration (FDA) approval in 2017. For some patients, this meant long term remission and even cures…
Published on September 21, 2022
Researchers at Friedrich-Alexander-University Erlangen-Nürnberg (FAU), in Germany have successfully cured several patients suffering from severe systemic lupus erythematosus (SLE) using genetically modified immune cells known as CAR-T cells. CAR T cells are immune cells taken from an individual patient and genetically engineered to express proteins known as CAR—chimeric antigen receptors—on…
Published on September 19, 2022
Rituximab, a monoclonal antibody therapy approved for treating rheumatoid arthritis and other autoimmune conditions, could be an effective first-line treatment for the autoimmune muscle condition myasthenia gravis according to a recent clinical study. In the trial, researchers from the Karolinska Institute in Sweden tested rituximab in a group of 47…
Published on August 17, 2022
A new study suggests a new way for hematologic oncologists to protect older patients from the risks of medication interactions. As part of the Older Adult Hematologic Malignancies Program, investigators from Brigham and Women’s Hospital and the Dana-Farber Cancer Institute studied the association between older patients with blood cancers who were…
Published on July 5, 2022
Research led by the Karolinska Institute in Stockholm has identified a urine biomarker that is lower in serious asthmatics than in individuals with a mild-to-moderate version of the inflammatory condition. Asthma affects 262 million people around the world, but is a very variable condition with the type and severity of…
Published on March 4, 2022
Immune-driven critical and chronic disease drug developer Endpoint Health announced earlier this week that a novel gene scoring model it developed has identified a molecular signature based on the expression of 15 genes that can classify certain patients’ immune profile and suggest a targeted approach to treating sepsis via a…
Published on December 27, 2021
The first study to evaluate outcomes of fully vaccinated cancer patients with breakthrough COVID-19 infections indicates they are at high risk for hospitalization and death. The study also appears to support previous observations that patients with hematologic malignancies are at greater risk for severe outcomes from the disease. Patients on…
Published on December 3, 2021
Purified IL-27 regulatory B cells (i27-Bregs) reduced symptoms in mouse models of multiple sclerosis (MS) and autoimmune uveitis, according to work by researchers at the National Eye Institute (NEI). The scientists report they have identified, isolated, and characterized a unique population of B cells that tamps down the immune system,…
Published on September 14, 2021
After the FDA rejected its bid to gain an emergency use authorization (EUA) for its drug lenzilumab as a treatment for patients hospitalized with COVID-19, Humanigen is vowing to continue pursuing approvals for the antibody treatment. The FDA stated in a letter that it was unable to conclude that the…