New research from the MRC Laboratory of Medical Sciences (LMS) has unveiled a groundbreaking method for discovering treatments for rare…
Browsing: Rare
The Rise and Fall of Sarepta Therapeutics’ Gene Therapy for Duchenne Muscular Dystrophy For Rachel DeConti and her son Jacob,…
Artificial intelligence (AI) is revolutionizing the field of medicine, particularly in the realm of rare disease research. A groundbreaking AI-powered…
The latest clinical trial led by the South Australian Health and Medical Research Institute (SAHMRI) is offering a ray of…
However, in people with alpha-1 antitrypsin deficiency, the faulty gene leads to a buildup of abnormal alpha-1 antitrypsin proteins in…
The discovery of a rare cell in the lining of the lungs has shed light on the organ-wide response necessary…
Fruit bats are known to carry Australian bat lyssavirus, but it is not known what species delivered the fatal bite.…
The Trump administration’s impact on gene therapy has been a topic of interest for researchers, advocates, and industry executives. Recently,…
The donor’s sperm carried a mutation that causes Li-Fraumeni syndrome or LFS, one of the most severe … More inherited…
Male Haemaphysalis longicornis (dorsal habitus). Credit: Journal of Medical Entomology, https://doi.org/10.1093/jme/tjaf040 An invasive tick species in Connecticut has been found…
