Experimental treatment reldesemtiv appears to reduce functional deterioration in patients with fast progressing amyotrophic lateral sclerosis (ALS), a new analysis of the FORTITUDE-ALS trial shows. The additional analyses were presented by the trial’s principal investigator, Jeremy M. Shefner, MD, PhD, a professor at the Barrow Neurological Institute…
The U.S. Department of Defense (DoD) has issued a two-year research grant totaling $758,121 to support a group of scientists from the University of Arizona Health Sciences Center for Innovation in Brain Science (CIBS) working on the development of RASRx1902, a potential treatment for amyotrophic lateral sclerosis…
The Chan Zuckerberg Initiative (CZI) has awarded I AM ALS a $453,000 grant to develop ways to connect amyotrophic lateral sclerosis (ALS) stakeholders and the public in the fight against ALS and other rare disorders. As part of CZI’s wider effort to accelerate investigations into rare diseases,…
Mutations in the annexin A11 gene (ANXA11), seen in familial amyotrophic lateral sclerosis (ALS) and frontotemporal dementia, disrupt the transport of key RNA molecules inside neurons, recent research shows. The annexin A11 protein is a molecular adaptor required for RNA molecules, essential for neurons’ function, to hitchhike on lysosomes…
In contrast to what is often assumed, new research suggests that clumps of the protein TDP-43 — a hallmark of amyotrophic lateral sclerosis (ALS) — actually protect nerve cells rather than harm them. If this finding confirmed in future studies, it could hugely impact how ALS and other neurodegenerative diseases…
The release of fragmented or dysfunctional mitochondria — a cell’s powerhouse — by immune and structural cells inside the central nervous system is a critical step that triggers neuron death and the progression of human neurodegenerative diseases such as amyotrophic lateral sclerosis (ALS), a study shows.
Next month’s annual conference of the National Organization for Rare Disorders (NORD) in Washington, D.C., couldn’t come at a better time, says Marshall Summar, MD, chairman of NORD’s board of directors. “The pace of discovery in rare diseases has gone from brisk to hypersonic,” Summar told Bionews Services, publisher…
After gathering input from patients, researchers, and advocates, the U.S Food and Drug Administration (FDA) has released new guidance on the development of therapies for amyotrophic lateral sclerosis (ALS). Among its recommendations, it is advising more communication with companies early on in the product development process, access to…
A detailed analysis of the communication network between nerve and muscle cells in turtles has shed new light on how movement is controlled and maintained. These findings on how motor nerve cells communicate may help scientists better understand the underlying mechanisms of amyotrophic lateral sclerosis (ALS) or spinal injury.
Kadimastem’s experimental cell therapy — AstroRx — may slow disease progression in patients with amyotrophic lateral sclerosis (ALS), according to interim results in a first group of patients treated in a Phase 1/2a study. AstroRx consists of functional healthy astrocytes — cells that support and protect neurons…
