If you have ever spent time sitting in your doctor’s office with a nurse hovering over you struggling to find a vein in your arm to take blood, you can…
Vicki and Fred Modell have spent years since their son’s death in the 90's advocating for more research and assistance to be provided to those with primary immunodeficiency disease. Their…
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Parkinson’s disease can be a hard disease to manage. Not only does it take a physical and emotional toll on patients and loved ones, but managing every day activities can…
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Major European and international kidney associations have joined in an awareness campaign to stress the urgent need to include patients with chronic kidney disease in clinical trials. In a recent…
As recently reported in CheckOrphan, a new drug for sickle cell disease has quickly ascended through the FDA approval processes in an accelerated review. A New Sickle Cell Drug Shroud…
Meet 6-year-old Grayson Kole Smith -- who after 36 surgeries and a grim prognosis has overcome the odds to live his life. Grayson was born with several serious medical issues…
Phenylketonuria (PKU) is a rare disease which causes phenylalanine (an amino acid) to build up in the body, leading to serious health complications. Since amino acids are obtained through the…
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