Donate to cure our dynamos!

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Your donations help support our patient organization. Our goal is to support expanding therapies for the DNM1 Developmental Epileptic Encephalopathy. The disease mechanism is fairly understood and multiple RNA silencing focused strategies underway… but more work is needed!
 

Research projects are translation focused, vetted by experts in the field and all donations go through Rare Village Foundation accounting operations which only pay out to official signed contracts, MTAs and invoices from our partnering organizations.
 

Given that a therapeutic path has been identified and having 4 programs funded generously by well-recognized institutions in the US, Canada and UK including 3 allele-specific RNA therapies in development, one base editing program and one allele-switching RNA therapy and that we have 2 humanized mouse models (the first ever for the disease) at Jackson Laboratories in Maine and 1 iPSC neuronal and retinal cells lines (the first ever outside of China) in Canada
 

where do we need help today is as follows:
 

1. Support to hire a part-time remote administrator to help with the below projects
 

2. Support to organize our first patient research and therapeutics conference in the US (we aim to minimize the cost by collaborating with another patient organization to host at the same time as their annual conference)
 

3. Support in building campaigns to increase awareness through carefully crafted marketing strategies.
 

3. Support in reaching out and finding patients worldwide and building our registry. We are currently collaborating with Citizen Health. Volunteers are working to find patients across the globe. We are working with volunteers in Japan and China to expand our network there. Our next project is to expand in LATAM.  We need support with translation.
 

4. Support a long-read sequencing campaign that aims to screen our patients. This will help us identify children with sequences or SNPs that may match the RNA therapies in development which will help expand the use of these therapies from n of 1 to "n of many".
 

5. Support generating iPSC cell lines for most recurrent variants (we have 2 variants that constitute the majority ~50% of cases)

6. Support Drug repurposing projects including screening through Unravel Bio’s platform and other organizations working in the field
 

7. Upcoming: Support a research project with a biotech company to help us identify an RNA strategy that can treat all variants that are amenable to splice-switching strategies. (Middle domain variants which are the most severe and have lowest survival rates)
 

8. Upcoming - Natural history studies - currently awaiting FDA grant feedback (we hope approval) through one of the top hospitals in the US and which if approved, your donations will help us support getting patients to the clinical site.
 

Should you have any queries, please do not hesitate to reach out to me at sali@dnm1epilepsy.com
 

Thank you in advance for your support
 

Sumaya
Rare dynamo mom