Rare Disease Science Challenge Submission Form

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Demographic Information

Background and Summary: Please describe the rare disease under study
(e.g. epidemiology, molecular basis, clinical presentation, available therapies, etc...).
Include a summary of research for this disease, and if appropriate, your own contributions. (750 word limit)

Please select donated services you'd like to apply for

View the full list of donated services

Platinum  Custom mouse model (Taconic) Human tissue functional assays (Biopta) Clinical Development Consulting (Charles River Laboratories) Genome Analysis Software (Ingenuity Systems) Primer design and qPCR analysis (DNA Software)

Gold Custom Zinc Finger Nuclease (Sigma Life Science) Expression analysis (Genemarkers) Assay development (donated by Cisbio Bioassays) Data collaboration software (Collaborative Drug Discovery) NGS Analysis (Novocraft Technologies) Bioinformatics software (BioDiscovery) Bioinformatics Consulting (BioVariance) TAQ Reagents (Sigma Life Science)

Silver and Bronze Sequencing services (Otogenetics) Chromosome methylation assays (Karyologic) Compound synthesis (Apertus Pharmaceuticals) Plasmid acquisition (Addgene) Chemistry consulting (SARmont) Custom antibodies (Antibodies Inc) Rare disease consulting (Three Pillars Biotechnology)

View the full list of donated services

Service Donation Prize Proposals: Based on the selected checkboxes above, please explain how you will make use of the selected service.
Please include scientific rationale, related prior research, and proposed next steps. (750 word limit per service selected)

Prize Proposal ($10,000 Prize): Describe the following:
-scientific approach and rationale for how you would utilize the cash prize for research
-how it would advance research for the rare disease under study
-estimate budget: how will the money would be used (750 word limit)

How did you hear about us?

By submitting this application, I acknowledge that I have read and agree to the guidelines, terms and conditions of this challenge.