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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November 13, 2025
Positive results at AHA Scientific Sessions, published in NEJM Evidence Continue Reading
October 15, 2025
Grant award provides MD/PhD candidates with funding and expertise to translate promising scientific discoveries into medicines Continue Reading
September 10, 2025
In ‘A Conversation on Courage,’ Lord Cameron and Morgan Stanley’s Andy Saperstein discussed philanthropy as a catalyst for rare disease innovation Continue Reading
July 20, 2025
Published in Good News NetworkEleven years of cancer research has proven fruitful for a scientist at Johns Hopkins who uncovered a new tumor-suppressive response that could lead to novel therapies targeting hard-to-treat cancers.The new study, funded in part by the National Institutes of Health and the National Cancer... Continue Reading
July 09, 2025
Experimental Compound Offers Potential Treatment for Rare, Often Fatal, Childhood Disease Continue Reading
July 09, 2025
Decreased brain levels of coenzyme Q10 (CoQ10), an endogenously synthesized lipophilic antioxidant, underpin encephalopathy in primary CoQ10 deficiencies and are associated with common neurodegenerative diseases and the ageing process. Continue Reading
June 24, 2025
Harrington Discovery Institute and the American Society for Clinical Investigation seek nominations to recognize an outstanding achievement by a physician-scientist Continue Reading
June 19, 2025
Enzymes that regulate oncogenic protein function through the addition of post-translational modifications (PTMs) represent promising targets for the development of cancer therapeutics. Continue Reading
June 04, 2025
The former prime minister has called for children admitted to paediatric care to undergo genome sequencing as part of a drive to cure 40 rare diseases Continue Reading
June 03, 2025
Drug development grant will advance 10 physician-scientists’ discoveries toward the clinic Continue Reading