EMERYVILLE, Calif. – Metagenomi, a gene editing company, today announced that it has received an award from the Cystic Fibrosis Foundation to identify and evaluate novel gene editing systems for the development of therapeutics to treat cystic fibrosis. “Cystic fibrosis is a complex, chronic genetic disease and there remains a...
people News
DETROIT – Ryder Washington started kindergarten this year. The 5-year-old loves to dance, practice martial arts and is obsessed with the Statue of Liberty. A few months ago he was diagnosed with a rare disease which in most cases leads to leukemia. The child’s family is hoping someone will be able...
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....
“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.
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...
A little girl who suffers from a serious lung disease and nearly died as a baby is on the path to a miracle recovery. Isabella Siddall, five, from Sutton, was born eight weeks before her due date, with underdeveloped lungs that led her to suffer from pulmonary hypertension (PT) and...
Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced the launch of a disease awareness campaign for Alagille syndrome (ALGS), a rare cholestatic liver disease affecting one in every 30,000 births worldwide. The campaign seeks to broaden awareness and education of ALGS so that healthcare providers can more readily identify symptoms and...