The Trump administration’s impact on gene therapy has been a topic of interest for researchers, advocates, and industry executives. Recently,…
Browsing: Rare
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…
Alnylam Pharmaceuticals: Pioneering RNAi Technology in Rare Diseases Alnylam Pharmaceuticals, a biotech company with a focus on RNA interference (RNAi)…
A recent study has shed light on a genetic clue that may help explain why some children are born with…
Patients with severe dystrophic epidermolysis bullosa, or EB, suffer from a rare genetic condition that affects 1 in every 500,000…
Plasma-activated medium (PAM) has emerged as a potential treatment option for synovial sarcoma, a rare form of cancer, according to…
When Michelle Werner received the devastating news that her son Caffrey had been diagnosed with Duchenne muscular dystrophy (DMD) five…
Rare Disease Policy: Incentivizing Development in a Challenging Landscape In the realm of rare diseases, the economics of research and…
$4M investment for digital portal on rare cancer A cutting-edge digital portal dedicated to providing information on rare and less…
