EU Horizon Project
SIMPATHIC is the abbreviation for “Accelerating drug repurposing for rare neurological, neurometabolic and neuromuscular disorders by exploiting SIMilarities in clinical and molecular PATHology”. Our consortium for the EU-funded Project SIMPATHIC consists of 22 international partners spread across Europe and Canada, including patient organizations, patient training organizations, academic centers, research infrastructures, and companies.
Tiny TIM
We are developing a new approach to expedite the development of existing drugs for groups of patients with rare neurological, neurometabolic and neuromuscular disorders. Suppose that Tim (or Tina or them) is suffering from a rare neurological disease. From a tube of Tim’s blood or a small piece of his skin, we will obtain Tim’s cells and convert them into one type of brain cells (so-called Tiny-Tim’s) for further research.
Focus on several diseases
The novelty of this project is that we focus on several rare diseases at the same time, rather than on one in particular. If we observe a positive effect of a drug, we will launch a clinical study in a group of patients with different disorders, but similar clinical symptoms.
Holistic perspectives on patients
We view Tim in a holistic way: he is a unique individual and so much more than his rare disorder, a set of symptoms, let alone his converted cells (Tiny-Tim’s). His well-being and quality of life are fundamental in the entire study of finding new therapies.