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.

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.

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.

Drug repurposing gives existing medicines a second life, but only when the business model clears the path. At Orfenix, we structure Intellectual Property strategically to align investor incentives, guarantee patient access, and deliver sustainable impact. Discover how our IP approach protects innovation and connects commercial potential with public health outcomes.

Orfenix proudly supports the development of guanabenz for Vanishing White Matter—a rare, progressive brain disease in children. Learn how repurposed drugs and dedicated research offer hope.

PXE is a rare disease with serious impact on daily life. We spoke with Heleen van Loenen from the Dutch PXE patient organisation about patient experiences—and why Orfenix is working to develop a treatment.

Pharmaceutical innovation thrives on a balance between exclusivity and accessibility. Limited-time commercial exclusivity incentivizes research while ensuring broader patient access over time. Discover how these mechanisms shape the future of equitable healthcare.

Drug repurposing – also called drug repositioning or drug reprofiling – is the identification of new therapeutic uses for existing medicinal products.

Orphan drugs, which are developed to treat rare diseases, face unique challenges in clinical trials. While the incentives for developing orphan drugs, such as tax credits and market exclusivity, have encouraged pharmaceutical companies to invest in this area, the clinical trial process remains fraught with difficulties.