… hypocalcaemia type 1 (ADH1), a rare disease caused by a mutation in …

BridgeBio Pharma on Wednesday said another one of its experimental medicines met its objectives in a late-stage trial, this time in a rare endocrine disorder. The therapy, known as encaleret, met all of its primary and key secondary goals in the study of …

… . It also accelerates progress in rare disease, oncology and personalized medicine, where …

… therapies for millions living with rare diseases. Under the 2025 theme, … , and legislative advocacy for rare diseases through talks and panels featuring … DakshamA Health, Global Genes, Patientworthy, Rare Disease Diversity Coalition (RDDC), Rare Revolution …

… kind of thrown in the rare disease community, so I had no … to develop gene therapies for rare diseases after his son Michael was …

The entertainment world was heartbroken to learn last week of popular young actress, Isabelle Tate. She was just 23. Tate recently appeared in the pilot episode of “9-1-1” Nashville.” “We are deeply saddened and completely heartbroken to share that …

“We’re often told in healthcare to listen to our patients,” says Dr Rachael Purcell, who led the detective work. “I think this is a really wonderful example of that.” Infant botulism is caused by spores from the bacteria Clostridium botulinum. In a baby’s …

… -aged man suffering from a rare disease. According to the non-governmental …