Rare disease is a major – and growing – area of clinical research and drug development. Of the FDA’s 46 novel drug approvals ...

When outcome selection and prioritisation are defined early in development, the evidence base supports continuity from trial ...

The IPO, which saw 25 million shares sold at $16, in the middle of the $15 to $17 range predicted by the company, continues a ...

Released ahead of tomorrow's Rare Diseases Day, the report (PDF) says that people affected by rare conditions face "profound ...

With the CHMP affirmation, Moderna is on course to get EU approval for its fourth vaccine product and break new ground in ...

Boehringer Ingelheim has become the second company to get a product approved under the FDA's national priority voucher ...

According to committee chairman Rick Scott (R-FL), "inconsistent review practices, shifting standards, and redundant, ...

Market expansion in med derm cross over: Almirall’s Seysara (sarecycline) won NMPA approval for moderate to severe acne (≥9 years), with China commercial launch targeted for 2026 – relevant to ...

As Rare Disease Day approaches, the lessons from recent European launches are clear: success comes from disciplined operating models, disease‑centred engagement, pragmatic evidence strategies, and ...

Oxford-based Sitryx, which also has operations in Boston in the US, was founded in 2018 with seed funding from SV Health ...

A recent study found that up to 40% of trials might be untrustworthy due to flaws in design, improper data collection, and ...

Eli Lilly's oral GLP-1 agonist orforglipron was more effective than Novo Nordisk's recently launched Wegovy pill in a head-to ...