by Hellen Houlleberghs | Apr 14, 2025 | Blog
At Orfenix, our mission is to develop therapies for unmet medical needs. One such need is a treatment for pseudoxanthoma elasticum (PXE), a rare genetic disorder that significantly impacts patients’ lives. Heleen van Loenen, head of the Dutch PXE patient organisation,...
by Hellen Houlleberghs | Jan 9, 2025 | Blog
The main challenges encountered during clinical trials include patient recruitment and retention, but also regulatory hurdles and methodological complexities need to be overcome. Drug repurposing is one of the possibilities used by Orfenix that aims to ease the...
by Hellen Houlleberghs | Nov 21, 2024 | Blog
Understanding EMA scientific advice The European Medicines Agency (EMA) scientific advice procedure was introduced with the aim to improve communication between companies developing medicinal products (sponsors) and regulators to enable streamlining of the development...
