Why Clinical Trials Matter — Especially for Rare Diseases

Davina McCall struggled with perimenopausal symptoms. She urges women to learn more about how this time of their life could affect them, and not suffer in silence. You were 43 when perimenopause started. Did you recognise the symptoms? No. But it got to the point where, on live TV, I couldn’t remember Laurence Llewelyn-Bowen’s name … Continued

By building tailored partnerships with innovators and drawing on deep local expertise, a new European model is unlocking the potential of rare disease medicines and reaching patients across all 32 countries. Across Europe, patients with rare diseases are being let down,” admits Adam Plich, and it isn’t due to a lack of pharma innovation. The … Continued

Artificial intelligence (AI) holds great potential to advance rare disease research. Here’s how scientists and physicians are using AI to improve outcomes for people living with rare diseases. How is AI used in rare disease research? Dr Kaminski: AI encompasses a broad set of applications. Scientists and physicians are using sophisticated computer vision or wearable … Continued