The patient affected by a serious adverse event in Neurogene's clinical trial of Rett syndrome gene therapy has now died, but that won't cause the study to be halted. The FDA has allowed the company ...

Elon Musk's brain-computer interface (BCI) company Neuralink has been given the green light to test its device in tetraplegic patients in Canada – the first international trial of the technology. The ...

US President-Elect Donald Trump is reported to be considering the nomination of Johns Hopkins surgeon and writer Martin Makary to lead the FDA after he takes office in January. The rumour was ...

AstraZeneca's anticoagulant reversal agent Andexxa was given an accelerated approval by the FDA in 2018, but the company's plan to get that upgraded to a full approval is facing some pushback. At an ...

Jazz Pharmaceuticals has claimed FDA approval for its HER2-directed bispecific antibody Ziihera as a treatment for biliary ...

The creation of these ‘pharmacy deserts’ exacerbates health inequalities and hinders the ongoing effort to shift the focus of healthcare towards prevention. Supporting community pharmacies, then, is ...

The term "digital twins" has its origins in engineering and computer science, where it refers to a virtual replica of a physical object or system. Simply put, it is a sophisticated, dynamic, real ...

Welcome to a new pharmaphorum round-up. These monthly gleanings of industry mergers and acquisitions, and also partnerships, will aim to provide an overview of what’s happening when it comes to ...

A diagnosis identifies a patient’s health problem and is a key to accessing the care and treatment they need. A diagnostic error is the failure to establish a ...

Data – it’s everywhere! Especially in clinical trials, where an average trial of about 2,000 patients can expect to produce 3,000,000 points of data across labs, vitals, medications, and ...

Leela Barham uses the very latest data – running up to November 2023 – to take stock of NICE’s Single Technology Appraisal (STA) recommendations on orphan drugs versus non-orphans.

Novel therapies, often with expensive price tags and finite patient populations, require strategic new positioning if they are to have maximum positive impact. Here, Alpharmaxim’s William Hind ...