Regeneron Pharmaceuticals has recently announced the results of Phase 2 of their study of pozelimab, which is an experimental treatment for paroxysmal nocturnal hemoglobinuria. Current therapies for this condition…
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According to the UK’s National Institute for Health Research (NIHR), a new technology called machine perfusion (MP) has the potential to solve organ shortage, a major challenge in liver transplantation.…
Genetic Counselors are an incredible resource – they can help assess your risk of developing a condition, the risk of your children inheriting a condition, and what potential treatment or…
“I feel like I’m in a huge dream that nobody can get into,” Noemie explained, describing her weeks of intensive sleep during which she slept 20 hours a day, the…
I wanted to invite the invisibly disabled community to join the screening of ‘We Are Visible’, my film about people living with an invisible condition (Ehlers-Danlos syndrome) all around the…
Sometimes in moments of tragedy, there is a bright silver lining that helps us learn, and that's exactly what Amber Briggle was aiming to do. Back in October, Amber posted…
No healthcare professional wants to hear that a drug being studied in a clinical trial did not meet the endpoints. It means that after countless hours of hard work,…
The FDA has just provided Orphan Drug Designation to Hizentra for the treatment of adults diagnosed with chronic inflammatory demyelinating polyneuropathy (CIPD). This is a maintenance therapy which works to…
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