Parents know how miserable it is when their child is sick. When my son was diagnosed with a rare, (at that time incurable) metabolic disease in 2016, a world fell apart for me. However, I have since completely changed the perspective of my son and that of many children with this disease.
From forming a patient advocacy group, managing it, awareness and fund raising I got to an organisation that successfully initiates research and therapy development.
This blueprint can be used to help MANY families with a rare disease child.
We are a team of passionate biochemists, patient advocates and consultants and aim to professionalise rare therapy development as mediator between patients and researchers. This solution, an independent consultancy and brokerage, brings benefits to all stakeholders: Scientists keep research in focus, patients their families & companies their development. One of the most vulnerable groups needs help - we have the energy, the vision and the knowledge to help MANY on the way to a therapy.
To kick off this impact startup, we need your help - please donate for hiring our fist part-time scientist!