“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.
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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...
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...