EL PASO — Two-year-old Emmanuel Gonzalez felt the sun on his face for the first time in weeks Sunday. He has a rare immune system disorder and had to stay in his room at Providence Memorial Hospital while he got well enough to make his first trip down to the...
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It’s shortly after 5 a.m. when the phone rings, and on the line is a clearly anxious and worried parent. “Sierra is having a lot of problems tonight,” Shaylene Akery tells a CNN producer. “We have to take her to the hospital, but we still want to talk to you...
A team led by a University of Florida Health scientist last month landed what it believes is the first National Institutes of Health grant ever awarded specifically to study a rare neurological disease that affects the lives of children and their families.
Houston , TX — University of Houston Assistant Professor of Biology and Biochemistry Quentin Vicens has been awarded a $1.2 million grant from the National Institute of General Medical Sciences to unravel the mystery of Z-RNA – an enigmatic RNA structure within our cells that plays a critical role in...
The son of Vitaliy Svichynskyi and Maria Svichynska was nearly two months old when he suddenly started to lose the ability to move. The family soon learned their baby boy Dmytro was born with Spinal Muscular Atrophy (SMA) Type 1, the most severe type of the rare genetic disorder found in...
NEW YORK, NY — Parent Project Muscular Dystrophy (PPMD) and the World Duchenne Organization (WDO), along with WDO’s members and affiliates, proudly herald the United Nations marking a momentous milestone for the global rare disease community by officially designating September 7th as World Duchenne Awareness Day (WDAD), set to be...
Sunday, February 28, 2021, was Rare Disease Day. With so much focus on COVID-19 throughout 2020, it’s important to recognize the continued work done in rare disease drug development by sponsors and FDA throughout 2020. In addition, a number of policies implemented in response to COVID-19 are expected to have...
ZURICH, Switzerland – Antiphospholipid syndrome is a systemic autoimmune disease associated with persistent antiphospholipid antibodies (aPL). It can cause thrombosis and pregnancy complications as well as non-thrombotic manifestations such as cardiac valve disease. Classification is important to help identify people to take part in research and ensures that similar disease...
Minnesota nice is being proven all over again. A young man from Africa says he is proof of that as doctors in St. Paul rebuild not only his face but his entire future. Even for skilled specialists, the surgical case on the table before them is a challenge. “It’s kind...
Houston, Texas – A four-year, $3.4 million grant to investigate molecular mechanisms and therapeutic treatments for acute respiratory distress syndrome (ARDS) has been awarded to UTHealth Houston researchers by the National Heart, Lung, and Blood Institute, part of the National Institutes of Health. The study led by principal investigators Holger Eltzschig,...