We’re committed to supporting patients in every way we can.
Who we are
Patients are at the heart of everything we do. We focus on providing treatments and services for patients with neurological, rare metabolic, and rare immunological conditions, most of which are genetic conditions.
Some of our patients are diagnosed with a rare disease at birth, others are affected later in life, and some can spend years trying to get diagnosed. No matter what stage our patients or their caregivers are at, we strive to provide them with the support they require because every single patient matters.
Our mission
To help and support patients and their caregivers with their needs or unique challenges, every single day.
Making a change
We aim to offer patients something they might not be able to get elsewhere. This includes improving the form the medication comes in, such as:
1. Making it smaller.
2. Making it temperature stable.
3. Upgrading it to a dissolvable tablet.
It also includes providing individualised services they might not be able to get with other treatments, such as:
1. Nutrition support through our Registered Dietitians.
Above and beyond
Where we can, we go above and beyond for our patients. Whether that is personally ensuring medication is on-site at a hospital for a newborn baby diagnosed with a rare disease, interacting with patients and their families at camps organised by rare disease patient advocacy groups, or presenting at meetings of our board of directors.
We’re passionate about helping people and making a positive difference, irrespective of their ability to pay for treatment. That’s why we’re proud to offer a Free Goods Programme for patients in countries where it is unlikely that they will have the means to pay for rare disease medication.
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