Innovative Support Services for Rare Diseases Grant
Granted by the Genetic Alliance
Details
Amount
Duration
Number of grants
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Eligibility
- Your organization must have been a non-profit for at least 2 years old
- Team size must be at least 3 members
- Must provide a report every 6 months
- Your organization must be compliant with the latest standards
Proposal Requirements
- Completed application form
- Innovative service proposal
- Detailed budget
- Proof of non-profit status
- Letters of support from patient groups
Supporting Materials
This study explores the potential of CRISPR-Cas9 gene editing technology in treating Myhre Syndrome. The findings indicate significant improvements in genetic correction and symptom management.
This research focuses on novel therapeutic strategies for SMOC1-related disorders. The results highlight the efficacy of targeted treatments in improving patient outcomes and quality of life.
The study investigates the effects of GPX4 deficiency on neuromuscular function. Findings suggest that supplementation with specific antioxidants can mitigate the impact of GPX4 mutations.