<div>The MED13L Foundation, along with the CURE MED13L research initiatives, announced that May 13th is now officially designated as “World MED13L Day.” We have launched a fundraising campaign to raise $1 million towards developing therapies for the single-gene disorder. The MED13L Foundation has outlined an initial milestone fundraising goal of $10,000 for the month of May, with a target of $250,000 raised by October 1, 2022.
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<div>MED13L is a recently identified single-gene disorder that is a leading cause of autism and intellectual disability. Other symptoms of MED13L include seizures, severe impairment of speech, motor developmental delay with poor muscle tone and ataxic-like movement, cardiac abnormalities and increased risk of childhood leukemia.
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<div>One of the initial goals of the MED13L Foundation was to connect with families around the world to better understand this syndrome. Today, MED13L Moms, Dads, and Caregivers are leading the charge.  We are pursuing the longer-term goal of advanced gene-based therapies.  The work we are doing has the potential to impact other syndromes beyond MED13L.  We are currently collaborating with researchers around the world to help facilitate a variety of research initiatives including:
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<div>✅ Mouse models. We have identified existing models in academic labs. We also applied to Jackson Labs Center for Precision Medicine for the development of mouse models. They have generously agreed to develop multiple mouse models on a pro bono basis, including humanized mice towards the pursuit of gene therapies.
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<div>✅ Dozens of MED13L cell lines, induced pluripotent stem cells (iPSC’s) and neurons.
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<div>✅ Diagnosis codes. Leading law firm Hogan Lovells has generously donated its time to help MED13L get an official ICD-10 diagnosis code, with benefits for identifying patient population, educating physicians, and reimbursement.
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<div>✅ The development of a patient registry and natural history database in order to facilitate clinical trials, as well as an online Cure-MED13L community forum.
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<div>In addition, the MED13L Foundation has partnered with Rarebase, a public benefit precision medicine company that leverages cutting-edge technology and biology to discover and develop treatments for the millions of people worldwide living with a rare disease.  The Rarebase Function™ platform combines leading-edge artificial intelligence and high-throughput assay systems to identify existing FDA-approved drugs that may improve MED13L and that could potentially be repurposed for MED13L patients in the near term. For more information about Rarebase, visit <a href="http://www.rarebase.org">www.rarebase.org</a>.<br /><br />To further establish the connection between MED13L families and the scientific community, we are partnering with Simons Searchlight <br />and holding a conference in August 2022 in Baltimore, Maryland. <br /><br />This one-million-dollar fundraising campaign has kicked off with the generous donation of a $100,000 matching grant by a relative of a MED13L family member.  We are humbly requesting help from around the world to support our efforts and make our goals a reality. Your donation will make a huge difference in the lives of many. Please know that we have outlined a clear path on how your donation will help to advance our research goals and will be meaningfully allocated. <br /><br />About CURE MED13L: Cure MED13L is an initiative of the MED13L Foundation, a 501(c)(3) non-profit organization founded in 2017, dedicated to supporting families with MED13L and to finding a cure.  MED13L is a rare disease that was first identified in 2013 and is a leading cause of autism and intellectual disability and includes other symptoms.  For more information, please visit the MED13L Foundation website or the Cure MED13L community forum. CURE MED13L (mn.co)<br /><br />MED13L Foundation website @ <a href="https://www.med13l.org" target="_blank" rel="noreferrer noopener">www.med13l.org</a>
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<div>CURE MED13L Forum @ <br /><br />
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<div>Please like and share our social media accounts @med13lfoundation
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merry campbell

Randall Borek

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1 Million For MED13L Fundraising Campaign

1 Million For MED13L Fundraising Campaign

Still $978,279 to go. Help us build momentum.