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Patients with ‘blinding’ headaches known as idiopathic intracranial hypertension (IIH) could be treated with an injectable peptide used for type 2 diabetes, a new trial has found. The study was led by a team of neurologists from the University of Birmingham and University Hospitals Birmingham, and published in the journal Brain. It reports on a […]

New data suggest that AKL Therapeutics’ investigational oral osteoarthritis (OA) drug APPA may have a dual effect on age-related cellular senescence. Senescence, a process in which cells stop dividing, plays a major role in the development of many age-related diseases, including OA. The study, using a human chondrocyte (cartilage-forming) cell line, was carried out at […]

New findings suggest that the SARS-CoV-2 virus damages an important immune-cell response. The quality of CD8+ T cell response to two doses of the Pfizer-BioNTech Covid-19 vaccine was considerably lower in people with prior SARS-CoV-2 infection compared to people without prior infection, the US study found. In addition, the level of this T cell was […]

A new approach that delivers a ‘one-two punch’ to help T cells attack solid tumours is the focus of a preclinical study by researchers from the Perelman School of Medicine at the University of Pennsylvania. The findings showed that targeting two regulators that control gene functions related to inflammation led to at least 10 times […]

Scientists have discovered why breast cancer cells that have spread to the lungs may ‘wake up’ following years of sleep – forming incurable secondary tumours. Patients with oestrogen receptor positive (ER+) breast cancer – the most common type – have a continued risk of their cancer recurring in another part of their body for many […]

For the first time, researchers have developed an mRNA vaccine that is 100% effective against a type of bacteria that is lethal to humans, paving the way for effective vaccination against antibiotic-resistant bacteria. The study, conducted in an animal model, demonstrated that all treated animals were fully protected against the bacteria. According to the researchers, […]

The US Food and Drug Administration (FDA) has cleared Intellia Therapeutics’ Investigational New Drug (IND) application for NTLA-2002 for the treatment of hereditary angioedema (HAE).  Intellia is a clinical-stage genome editing company focused on developing potentially curative therapies using CRISPR-based technologies.   The decision will enable the company to include the United States in the global […]

CAR-T cell therapy is effective in mice with ovarian cancer, according to a study published in The Journal for ImmunoTherapy of Cancer.   The study authors, who work at Karolinska Institutet, hope that the discovery will pave the way for a clinical trial to see how effective the treatment is for women with the disease.   “This […]

The European Commission and the UK Government have reached a political agreement that they hope will address the challenges faced by citizens and businesses in Northern Ireland (NI) following Brexit. The Windsor Framework, which focuses on a variety of issues, including supply of medicines, constitutes a comprehensive set of joint solutions aimed to ensure lasting […]

Rare diseases are often overlooked for funding and research, but with various incentives and priority review programmes now in place, many companies are beginning to explore these unusual but often devastating conditions. This Rare Disease Day we provide an overview of the progress that has been made in a range of rare diseases so far […]