by Max Verhage | Oct 6, 2025 | Blog
Cystinosis is a rare hereditary disease which leads to accumulation of cystine in the lysosomes causing organ failure. The disease manifests in all organs, but the kidneys and the eyes are affected first. Even though for cystinosis there is a therapy available,...
by Barbara Van den Aarsen | Sep 12, 2025 | Blog
Investigator-initiated clinical trials trials play an important role in advancing medical knowledge, exploring novel uses for existing therapies, and addressing clinically relevant questions that industry trials may overlook. However, these trials come with unique...
by Vincent Van der Wel | Aug 11, 2025 | Blog
Developing a medicine that is accessible for patients remains a big challenge, especially in areas like rare diseases or drug repurposing. Every day new papers are published with potential treatments in the scientific literature, but the path from lab to clinic is...
by Ivo De Nooijer | Jul 14, 2025 | Blog
Repurposing existing drugs for new indications has emerged as a powerful strategy in modern drug development. Yet, the business models that underpin successful repurposing ventures remain diverse and highly dependent on how intellectual property (IP) is managed. In...
by Hellen Houlleberghs | Jun 17, 2025 | Blog
A promising rare disease treatment supported by Orfenix. At Orfenix, we are dedicated to turning academic breakthroughs into life-changing therapies through efficient and socially responsible drug development. One of our key projects focuses on guanabenz, a promising...
by Barbara Van den Aarsen | May 22, 2025 | Blog
Clinical trials play a vital role in developing new treatments and medications. However, when children are involved, the process becomes significantly more complex. Children are considered a vulnerable population, requiring careful ethical, regulatory, and...
