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...
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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,...
A Valley boy diagnosed with a rare and fatal childhood disease is now being allowed access to an experimental drug. Denise and Rickey Miller’s 21-month-old son Woodrow has Niemann-Pick Disease Type C. It’s a form of childhood Alzheimer’s that eventually takes away the child’s ability to walk, talk, swallow, and breathe. For...
Blacksburg, Virginia – A team of Virginia Tech researchers was awarded nearly $2 million from the National Institute of Diabetes and Digestive and Kidney Disease, part of the National Institutes of Health, to explore novel approaches for treating Type 2 diabetes and obesity. Type 2 diabetes affects more than 38.4...
In a touching turn of events, the Vodafone Ghana Foundation has made a profound impact on the life of Oleki Gaduga, a 12-year-old girl from Ada. Oleki was born with Infantile Blount Disease, a rare disorder that causes leg bowing and difficulty walking. Oleki had been suffering from severe pain...