Forest Ray PhD,  —

Scientists have identified molecules that enable them to study and manipulate a specific type of cell present in the central nervous system, called glial cells, at the junctions where nerve cells communicate with muscles. Understanding how these cells operate at such sites, called neuromuscular junctions or synapses, has…

Distinct RNA patterns in a person’s blood may signal the presence of amyotrophic lateral sclerosis (ALS), a study reports. Researchers, led by Majid Hafezparast, PhD, a professor of molecular neuroscience at the University of Sussex in the U.K., discovered that certain types of non-coding RNA (ncRNA), a…

The Alexion Charitable Foundation has awarded $1.1 million in grants to programs that support those with rare diseases during the COVID-19 pandemic, the organization recently announced. The grants will support activities that align with the foundation’s Rare Belonging focus, a set of funding priorities aimed at improving the…

The Black Women’s Health Imperative (BWHI) recently created a Rare Disease Diversity Coalition focused on reducing racial disparities in the rare disease community. Getting a timely and accurate diagnosis for a disease that few people — sometimes even physicians — have heard of is challenging on its own merit.

Two bills making their way through Congress are intended to improve access to medical therapies for those most in need, and in particular, those with amyotrophic lateral sclerosis (ALS). Sen. Mike Braun, R-Ind., announced his plan to introduce a bill to hasten the regulatory process for making promising therapies…