MIDDLETON, Idaho — When Jake and Haley Comer of Middleton welcomed their new baby boy Ryder into the world, they were completely overjoyed. But, they quickly realized something might not be right. “The whole pregnancy was completely healthy, there were no signs of anything.” said Haley Comer, Ryder’s proud mom....
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LAUSANNE, Switzerland – Progression of Duchenne Muscle Dystrophy (DMD) can be delayed in mice by supplementing their diets with Urolithin A, according to new results reported today. The findings, published in Science Translational Medicine, raise hopes that new treatment options could one-day be developed for DMD, an uncurable genetic condition...
“The National Organization for Rare Diseases estimates that 1 in 10, or 10%, of Americans have a rare condition. So you have to start thinking, how rare is rare when you think of it cumulatively?” says Immune Deficiency Foundation President and CEO Jorey Berry.
MUNICH, Germany – Minaris Regenerative Medicine GmbH (“Minaris”), a leading contract development and manufacturing organization (CDMO) for the cell and gene therapy industry, will be the commercial manufacturer in Europe for Skysona™ (elivaldogene autotemcel, Lenti-D™), a product of bluebird bio, Inc. Today, the European Commission (EC) has granted marketing authorization for...
Charleston, SC – A team of researchers at the Medical University of South Carolina and Cincinnati Children’s has developed a sophisticated model for studying the diseased colon that could lead to the development of personalized treatments for colon-related diseases, such as cancer and inflammatory bowel disease (IBD). The researchers report...
College Station, TX – The first glucose self-monitoring system created in 1970 weighed three pounds, was initially designed only for physicians’ offices and needed a large drop of blood for a reading. Over 50 years later, researchers at Texas A&M University are working to create a fully injectable continuous glucose...
As someone being treated for a rare disease in Minnesota, I am experienced with prescription drug costs. Living with cystic fibrosis for decades now, its related pain, treatments and challenges, I can appreciate the need to lower costs for medications. The question is, what is the best way to do...
Philadelphia — Every cause needs a champion. That’s why Minnesota Twins outfielder Michael A. Taylor has launched a season-long home run challenge to support Uplifting Athletes and the rare disease community. Inspired by his sister Danielle, Taylor will use his platform as a Major League Baseball player to inspire hope...
CAMBRIDGE, Mass. & HAIFA, Israel – Minovia Therapeutics, a clinical-stage company focused on the development of cell-based Mitochondrial Augmentation Therapy (MAT) to treat mitochondrial diseases, announced today the appointment of a key member of Management for the Company’s next phase of growth. In anticipation of feedback from the FDA on...
Christine Falleti has spent much of her life combating the crippling effects of cystic fibrosis (CF). Now 34, she is painfully aware that she’s approaching the age when most people with CF die. Two friends with the disease already have. But last year she took part in a test of...