December 23, 2025
Rates of obesity and associated fatty liver disease have risen as the world increasingly adopts energy-dense diets and sedentary lifestyles. Cleveland research team has discovered a new enzyme that is required to make fat. Blocking the enzyme prevented weight gain and lowered cholesterol. A three-in-one drug is being...
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December 23, 2025
A conversation with Matthew P. Anderson, MD, Ph.D., co-director, Oxford-Harrington Rare Disease Centre Continue Reading
December 22, 2025
CLEVELAND – For over a century, Alzheimer's disease (AD) has been considered irreversible. Consequently, research has focused on disease prevention or slowing, rather than recovery. Despite billions of dollars spent on decades of research, there has never been a clinical trial of a drug for AD with an outcome goal of... Continue Reading
November 20, 2025
Ten scientists selected for second annual award to pursue innovations in gene, RNA, small molecule and cell therapies to treat rare and ultra-rare diseases Continue Reading
November 14, 2025
Scientists are trying a revolutionary new approach to treat rheumatoid arthritis, multiple sclerosis, lupus and other devastating autoimmune diseases — by reprogramming patients’ out-of-whack immune systems. Continue Reading
November 13, 2025
Positive results of zalunfiban announced at AHA Scientific Sessions 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
Published in NatureAbstractDecreased 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,. CoQ10 supplementation does not increase CoQ10 pools in the... Continue Reading