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New RARE Toolkit “Searching For Answers: Contacting Biopharmaceutical Companies Effectively”

“I can do that?” This is the question…

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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
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Lisa with Adrenal Cortex Carcinoma, “I Had to Fight for My Treatment Plan.”
I was 35 years old when I was diagnosed with Adrenal Cortex Carcinoma–stage IV, metastasized to the lungs. I was recently divorced, a… Continue Reading
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Kickin’ Fightin’ and Strong: Klippel-Feil Syndrome and Me
I was diagnosed with Klippel-Feil Syndrome (KFS) shortly before my first birthday. At birth I had the ‘classic’ triad of… Continue Reading