Partnering with the rare disease community
At Orphalan, patients inspire our work and serve as our collaborators. We recognise that in the world of rare diseases, the patient and their family are often the leading experts, and our commitment is to listen, learn and incorporate the patient voice into our work.
This is how we collaborate with patients, caregivers and the wider rare disease community, at every stage of our work.
We actively seek out the lived experiences of those affected by rare conditions. By engaging with patient advocacy groups and community leaders, we aim to ensure our clinical development programmes reflect the real-world challenges patients face every day.
We do not just develop medicines for patients; we develop them with patients. This means involving patient experts in the design of clinical trials to help reduce participation burden and identify outcomes that are meaningful to the community.
Our engagement does not end with a prescription. We work to reduce geographic and financial barriers to treatment, providing educational resources and support systems intended to help patients manage their health.
Whether you are newly diagnosed, a long-term patient or a caregiver, here are the resources and people available to you.
Clear, accessible information about Wilson's Disease, West's Syndrome and other rare conditions we focus on.
Connect with patient organisations and communities near you, because no one should navigate a rare disease alone.
Practical guidance on taking your medicine safely, recognising side effects and what to do if you have concerns.
Whether you have a question, a concern or simply want to share your experience, our team is here to listen.
Partnerships with Patient Organisations
Orphalan is proud to collaborate with global and local rare disease organisations. These partnerships allow us to support disease awareness campaigns, contribute to patient registries (like the iWilson's Disease Registry) and advocate for policy changes that improve the lives of those living with unmet medical needs.
Through these collaborations, we amplify the voices of patients and caregivers, working together to create meaningful change in the rare disease landscape. Our partnerships are built on mutual respect, shared goals and a commitment to putting patients first.
Find a Patient Support GroupEthics and Transparency
Our interactions with patient communities are governed by the highest ethical standards and transparency. We respect the independence of patient organisations and ensure all collaborations are conducted with integrity, following global regulatory guidelines to ensure a clear boundary between support and promotion.
Every partnership is designed to benefit patients and the broader rare disease community, with full transparency in funding, objectives and outcomes. We are committed to maintaining trust through responsible and ethical engagement practices.
Answers to common questions, whether you are still waiting for a diagnosis or already living with a rare condition
Whether you are still going through tests or have recently received a diagnosis, this can be a difficult and uncertain time. Many people find it helpful to keep a simple record of symptoms, appointments and questions as they arise, to share with their healthcare team. Your healthcare team remains the best source of guidance on what may be most useful for your specific situation.
Some people find it useful to write down symptoms and questions beforehand, and to bring a family member or friend along for support. Asking your healthcare team to explain anything that is unclear is always welcome, whether you are working toward a diagnosis or discussing an existing one.
There is no single right way to approach this, and every family's situation is different. Some people find it helpful to share what they do know in simple terms, even while some questions remain unanswered. Healthcare teams may also be able to point families toward relevant support services during this time.
It is common to experience a range of emotions, whether navigating uncertainty before a diagnosis or adjusting to life afterwards. Some people find it helpful to talk things through with family, friends or others who understand what they are going through, while others prefer to speak with their healthcare team about how they are feeling. Healthcare teams may also be able to signpost appropriate emotional or psychological support services, as well as relevant patient communities, in your area.
Yes. A number of organisations support people and families at different stages, including those still awaiting a diagnosis as well as those living with a confirmed rare condition. A healthcare team or specialist centre may be able to signpost relevant organisations or communities suited to your situation.
This information is for general guidance only and is not a substitute for professional medical advice. Always consult your doctor or specialist about your individual care.