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...
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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...
Barcelona, Spain and Düsseldorf, Germany – Minoryx Therapeutics, a late-stage biotech company focused on the development of therapies for orphan central nervous system (CNS) disorders and Neuraxpharm Group (Neuraxpharm), a leading European specialty pharmaceutical company focused on the treatment of CNS disorders, today announce that the Marketing Authorization Application (MAA)...
Barcelona, Spain – Minoryx Therapeutics, a late-stage biotech company focused on the development of therapies for orphan central nervous system (CNS) disorders, today announces that the first patient in the TREE study has been dosed with leriglitazone. TREE is a phase 2a clinical study assessing the safety and efficacy of...
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...
HAIFA, Israel — Minovia Therapeutics Ltd, a clinical stage biopharmaceutical company advancing mitochondrial therapies for primary and secondary mitochondrial diseases, today announced that the U.S. Food and Drug Administration (FDA) has cleared its second Investigational New Drug (IND) application for MNV-201, an autologous hematopoietic stem cell product augmented with allogeneic mitochondria. The IND supports the initiation of a Phase II clinical trial of MNV-201 in pediatric patients...
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...