In Germany, over four million people suffer from one of the more than 8,000 known rare diseases. Cures are rare, and the industry often finds research unattractive because only a few patients need the drug that has been developed. Conventional animal-based research is not effective here, animals do not develop these rare diseases, animal "models" often do not replicate the reactions in the human body due to species differences.

For developers of multifluidic systems or, for example, organ or human-on-a-chip models, working with patient cells opens up opportunities. These technologies have the potential to identify individual treatment options for rare diseases. Examples include in vitro models for research into ALS, myasthenia gravis with organ-on-a-chip and the investigation of tuberous sclerosis and Duchenne muscular dystrophy.

Encouraging results come from an American team from Florida that has successfully treated with an antibody developed by Sanofi in a microfluidic system. "These results have already been accepted by the US FDA," says Dr. Christiane Hohensee from InVitro+Jobs, the science platform of the Federal Association, "It is a hopeful step towards animal-free drug development.

In Germany, there is even a special BMBF funding program to support a limited number of transnational, ambitious, innovative and high-quality studies on the natural course of rare diseases.

Further information can be found at:
https://www.invitrojobs.com/index.php/en/news/news-archive/item/5603-florida-new-neuropathy-model-on-chip-for-drug-studies-of-rare-diseases
https://www.invitrojobs.com/index.php/
https://www.invitrojobs.com/index.php/en/news/news-archive/item/5906-bmbf-is-funding-in-vitro-studies-on-rare-diseases