Spinal Muscular Atrophy (SMA) causes progressive muscle weakness and, without treatment, can limit life expectancy to just ...

A Marion woman is celebrating a victory for those suffering from rare diseases across the state after securing a proclamation from Governor Andy Beshear declaring December 2025 as Brooke-Spiegler Synd ...

Several children in the UAE, facing developmental delays and muscle weakness underwent standard genetic tests only for the ...

An immune-related gene has now been tied to a rare inherited neurological disorder, revealing an overlooked pathway in the nervous system.

GeneDx Holdings Corp. (NASDAQ: WGS) is one of the most promising stocks under $100 to buy. On March 10, GeneDx and Zevra ...

"Squawk Box" co-host Becky Quick launched CNBC Cures in January, the network's initiative to raise awareness for rare ...

More than 5,000 shelved drug candidates were discontinued for business reasons, not safety ones. A new push aims to match ...

Lo, Ph.D., Professor at the MIT Sloan School of Management and a member of n-Lorem’s board of directors, have published a commentary in Nature Biotechnology that proposes more economically feasible ...

Ava, Olivia, and Landon Langenhop were all born with a rare and life-threatening immune disorder called severe leukocyte adhesion deficiency-I, or LAD-I. This rare genetic condition makes them at risk ...

Jack Arneson, born Jan. 26, has both Ebstein's anomaly, a congenital heart defect, and Sotos syndrome, a rare genetic ...

Mirecule, Inc., a leader in Antibody RNA Conjugate (ARC) therapeutics targeting rare neuromuscular diseases, today announced an expansion of its strategic collaboration with Sanofi. This collaboration ...

Blindness and deafness in some infants and sickle cell are among the conditions cured in recent gene therapy trials.