Bringing medicine back to life?
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What We Do.
We turn great ideas into accessible medicines. To do that we work with academics to turn their ideas into reality and in a societally responsible manner. For us, this means building a sustainable business while ensuring patient access. Explore our portfolio to see how we realize that ambition.
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Reformulating For Cystinosis
Repurposing For PXE
Repurposing For VWM
Academic Partners.
Insights.
Cystinosis: the past, present and future
Cystinosis is a rare hereditary disease that affects multiple organs, of which the kidneys and eyes are affected first. In conversation with Irene Kinds from the Cystinosis Patient Organisation Vlaanderen and The Netherlands, and mother of a 16-year-old living with cystinosis, we discuss what daily life with the disease looks like, why early diagnosis and adherence to therapy matter, and how new treatments could change the future for patients worldwide.
Things to get right when conducting investigator-initiated clinical trials
Investigator-initiated trials drive innovation but require careful planning. Success depends on clear protocols, compliance, data quality, safety, and transparency. This ensures credible, clinically significant results that complement industry research and advance patient care.
How does a public-private-partnerships in drug development work?
Developing a new medicine is a long, costly journey—especially in rare diseases or drug repurposing—where traditional funding models often fall short. Public-Private Partnerships (PPPs) offer a powerful alternative, bringing academics, industry, and patient groups together to share risks, combine expertise, and keep patient access at the heart of development.
Contact Orfenix.
For more information or to explore partnership opportunities, feel free to reach out to us anytime!
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