The Global Genes 2018 RARE Patient Advocacy Summit – one of the key events in the rare disease world – wraps up today. As the largest worldwide gathering of rare disease patients, advocates, and thought leaders, the summit brought together over 100 experts, who led educational sessions in specialized fields. Among them was Ethan Perlstein, Perlara’s founder and CEO, who led a presentation on “Animal Models and 3D-Engineered Tissues.” If you missed the presentation, this post can fill you in on what Ethan discussed in his 2018 Global Genes presentation.

 

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The Global Genes 2018 RARE Patient Advocacy Summit synopsis of Ethan’s presentation:

Animal models and 3D-engineered tissues play a vital role in developing therapies for rare disease. Patient advocates need to understand how these technologies help provide a way to study rare diseases and test potential therapies for safety and efficacy. Different animal models have different advantages, and sometimes much can be learned from very simple models. However, animal models are not human tissues, which can present challenges. Engineered tissues can help drive treatments because they can model the structure and function of human tissues and complement animal models.”

 

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“The goal of the talk was to provide patient advocates a basic fact sheet for each of the model organisms – from yeast to nematodes to flies to fish to mice – to help answer questions such as: what fraction of genes in the fly genome are evolutionarily conserved in humans? How long does it take, on average, to genetically engineer a nematode patient avatar? We also felt it was important to include estimated budgets for model making and drug screening activities in each of the organisms so that foundations know how much it costs to push research forward.” 

Ethan Perlstein, CEO, Perlara PBC

 

Perlara’s 2018 Global Genes presentation

 

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Want to know more about how Perlara collaborates with families, patient advocacy groups and researchers to find cures for your disease, and potential cures for other diseases? Learn what’s in a PerlQuest™ and how you, or your organization, can launch a PerlQuest. Remember to share some information to get things started. Let’s PerlQuest together!