Rare Diseases & Genetics

8+ hour, 34+ min ago   (280+ words) The Times of India Ahmedabad: A national-level meeting on “inflammatory myositis”, an autoimmune disease that affects muscles, was organised at NHL Municipal Medical College in Ahmedabad, bringing together nearly 175 doctors, researchers, trainee physicians and students from across the country....

12+ hour, 18+ min ago   (688+ words) People who carry a rare inherited mutation in the EGFR gene are 25 times more likely to develop lung cancer than non-carriers, with the excess risk increasing to 62-fold among nonsmokers, making it one of the strongest risk factors for any…...

14+ hour, 3+ min ago   (306+ words) As biotechnology continues its march toward revolutionary treatments and cures for complex medical conditions, the daily realities for people living with rare diseases and their families can dampen hope. Worldwide, the relentless cycle of caregiving, stress and hypervigilance only deepens…...

15+ hour, 14+ min ago   (597+ words) PR Newswire NEW YORK, Sept. 23, 2026 /PRNewswire/ -- Apertura Gene Therapy, a biotechnology company developing next-generation AAV capsids for delivering genetic medicines, will provide Lexeo Therapeutics, Inc. (Nasdaq: LXEO), a clinical stage company focused on reshaping the path of genetic diseases with…...

15+ hour, 24+ min ago   (384+ words) £140,000 has already been raised towards the fund to help Tate(Image: Tammy McDaid) Life-changing gene therapy which a five-year-old boy with childhood dementia needs to stop his terminal condition progressing has been approved in the US, as his mum continues…...

11+ hour, 40+ min ago   (577+ words) Rare diseases are defined individually by their scarcity, but collectively they represent one of the largest burdens of unmet medical need in modern medicine....

17+ hour, 19+ min ago   (324+ words) BMPR2 acts like a receiver on the surface of cells. Normally, it responds when a signaling molecule arrives, then helps pass carefully timed instructions into the cell. Those signals are part of the bone morphogenetic protein, or BMP, pathway, which plays…...

20+ hour, 52+ min ago   (187+ words) September 23, 2026 - AstraZeneca today announced a new five-year phase of its flagship Young Health Programme (YHP) in partnership with UNICEF and 60 non-governmental organisations (NGOs), extending its investment in youth health equity to over $100m since the YHP's inception in 2010. Catherine Russell, UNICEF…...

23+ hour, 14+ min ago   (177+ words) Fibromyalgia “is a multifaceted syndrome,” for patients, presenting with a range of symptoms from pain to fatigue, cognitive impairment, and body discomforts found in other conditions, such as irritable bowel syndrome. For clinicians, the plethora of waxing and waning symptoms…...

19+ hour, 22+ min ago   (644+ words) The Asian tiger mosquito, Aedes albopictus, is one of the most successful invasive species on the planet, and in China it stands as the principal vector of dengue and other arboviruses that threaten hundreds of millions of people. A new…...