December 03, 2024
Inherited SP-B deficiency is an ultra-rare monogenic cause of fatal respiratory distress syndrome in newborn infants with few, if any, options for long-term survival Highly encouraging preclinical data highlight the possibility of lifelong treatment from a single administration of AVG-002 with preparations for clinical...
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November 15, 2024
Oxford, UK – 15th November 2024 – (or ‘the Company’, www.alveogene.com), a company focused on transforming rare respiratory disease outcomes using inhaled gene therapy, announces that it has been granted a Rare Pediatric Disease Designation (RPDD) by the US Food & Drug Administration (FDA) for AVG-002, its novel... Continue Reading
October 24, 2024
10 scientists have been selected for OHC’s first annual award to advance academic discoveries into clinical practice for rare diseases Continue Reading
October 15, 2024
Lord Cameron will lead international efforts to drive the OHC’s mission of delivering 40 new therapies for rare diseases into clinical trials over the next ten years Continue Reading
September 26, 2024
Grant award provides MD/PhD candidates with funding and expertise to translate promising scientific discoveries into medicines Continue Reading
September 24, 2024
The Symposium explores how cutting-edge research, innovative treatment strategies, and collaboration is driving the development of effective therapies for rare diseases. Continue Reading
September 17, 2024
Progression of acute TBI to chronic neurodegeneration is a major health problem with no protective treatments. Time sensitive inhibition of acutely elevated mitochondrial fission may present a way to protect patients from chronic neurodegeneration. Continue Reading
September 11, 2024
In her position on OHC’s Advisory Council, Baroness Blackwood will contribute to advancing OHC’s mission to drive life-changing discoveries in rare disease, with a particular focus on addressing critical unmet needs in the UK. Continue Reading
September 10, 2024
Now, University Hospitals (UH) and Case Western Reserve University researchers have moved a step closer to finding answers in a study recently published in Cell Reports Medicine. Continue Reading
July 23, 2024
Harrington Discovery Institute and the American Society for Clinical Investigation seek nominations to recognize an outstanding achievement by a physician-scientist Continue Reading
July 16, 2024
There’s only one U.S. Food and Drug Administration-approved therapy for an inherited retinal disease, and dozens of retinitis pigmentosa (RP) genes for which no therapy is available. Continue Reading