From Our Affiliates
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Lab-Developed Intestinal Organoids Form Mature Human Tissue In Mice

Researchers have successfully transplanted…

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NBC News Reports: California Boy Raises $800,000 to Fight Friend’s Rare Disease
Lora Pournazarian rarely sleeps through the night. A bundle of nerves, Lora is up every few hours — eyes fixed on the alarm clock,… Continue Reading
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UNMC Researcher Receives $3.3 Million Grant to Study Rare Diseases That Affect Children
University of Nebraska Medical Center researcher, William Rizzo, M.D., has received a five-year, $3.3 million grant to study 10 rare… Continue Reading
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BioPontis, Reborn as Nonprofit, Takes On Rare Disease Drugs
Of the more than 7,000 rare diseases that each affect just a sliver of the population, an estimated 95 percent of them have no treatment.… Continue Reading
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Balancing Act Premieres New Segment on Paroxysmal Nocturnal Hemoglobinuria Tomorrow Morning
(PNH) Paroxysmal Nocturnal Hemoglobinuria is a deadly life-threatening disease with devastating consequences.  35% of patients die within… Continue Reading
Adam L. Boxer, M.D., Ph.D., who leads the RDCRN-supported Frontotemporal Lobar Degeneration (FTLD) Clinical Research Consortium, is exploring a group of progressive brain diseases that damage brain networks controlling behavior, cognition and movement. This image illustrates severe brain tissue loss over one year for patients with two types of FTLD: behavioral variant frontotemporal dementia (red-yellow) and progressive supranuclear palsy (yellow, orange and blue). (S. Dutt, H. Rosen, A. Boxer Photo)
NIH Funds Research Consortia to Study More Than 200 Rare Diseases
Physician scientists at 22 consortia will collaborate with representatives of 98 patient advocacy groups to advance clinical research and… Continue Reading
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October 14th is Global Food Protein Induced Enterocolitis (FPIES) Day!
The FPIES Foundation has established October 14th as Global FPIES Day. FPIES (Food Protein-Induced Enterocolitis Syndrome) is a rare type… Continue Reading
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New Study on CAID Syndrome Links Heart and Gut
Physicians and researchers at CHU Sainte-Justine, Université de Montréal, CHU de Québec, Université Laval, and Hubrecht Institute have… Continue Reading