February 28, 2026
Op-Ed Authored by Lord (David) Cameron, former UK Prime Minister and Chair of the OHC Advisory Council published in The Daily Telegraph on Saturday, Feb. 28, 2026 (Rare Disease Day) Rare diseases are not actually that rare. Collectively they affect 500 million people around the world. Some, such as Huntington’s disease...
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December 11, 2024
An off-the-shelf drug shows promise as a low-cost topical treatment for psoriasis. New findings highlight benzamil’s potential to reduce inflammation and skin cell overgrowth without steroids or immune suppression. Continue Reading
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 Continue Reading
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