Marta Figueiredo, PhD, managing science editor —

The first wave of COVID-19 in Europe severely disrupted access to care and raised stress and anxiety in people with rare diseases, negatively affecting their health and well-being, according to a survey conducted by Eurordis-Rare Diseases Europe. “People living with rare diseases in Europe have found themselves caught as collateral…

BrainStorm Cell Therapeutics’ cell-based therapy NurOwn failed to significantly slow disease progression in people with rapidly progressing amyotrophic lateral sclerosis (ALS), top-line data from a Phase 3 trial show. The therapy, however, did result in clinically meaningful responses in a pre-specified group of patients with early ALS…

Bristol Myers Squibb has entered a five-year research collaboration with the machine learning startup insitro to discover and develop new therapies for amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). Machine learning is a form of artificial intelligence that uses algorithms to analyze large amounts of data, learn…

MND-SMART, a pivotal platform trial designed to simultaneously test multiple treatment candidates in people with amyotrophic lateral sclerosis (ALS) and other motor neuron diseases, has opened its third clinical site in Scotland. This type of platform trial, distinct from typical clinical trials that test a single treatment…

Treatment with AMX0035, Amylyx’s experimental oral therapy, significantly extends the lives of amyotrophic lateral sclerosis (ALS) patients with rapidly progressing disease, according to a long-term survival analysis of the CENTAUR Phase 2/3 trial. The new data were reported in a study titled “Long‐Term…

The National Institutes of Health (NIH) has awarded a $1.9 million grant to University of Missouri (MU) researchers to study ways of preserving essential functions like swallowing and breathing in people with amyotrophic lateral sclerosis (ALS) and other motor neuron diseases. This four-year grant was awarded to…

Amylyx’s experimental oral treatment AMX0035 safely and effectively slows functional decline in amyotrophic lateral sclerosis (ALS) patients with rapidly progressing disease, according to full data from the CENTAUR Phase 2/3 trial. Only changes in upper limb strength were significantly different from those seen in placebo-treated patients, the data show. Meanwhile,…

Pharnext’s investigational oral combination therapy PXT864 prevents degeneration of motor neurons and their point of contact with muscle cells, as well as the buildup of toxic TDP-43 protein aggregates — all hallmarks of amyotrophic lateral sclerosis (ALS) — in cellular models of the disease. The data…

BrainStorm Cell Therapeutics’ cell-based therapy NurOwn increases the number of immunosuppressive cells and lessens inflammation, according to a small preclinical study. These immunoregulatory effects, along with NurOwn’s neuroprotective and neuroreparative properties, may be key to halt the progression of amyotrophic lateral sclerosis (ALS) and other neuroinflammatory diseases, including…

Orion Corporation’s oral therapy levosimendan (ODM-109) failed to preserve lung function and overall functionality in people with amyotrophic lateral sclerosis (ALS), data from the Phase 3 REFALS clinical trial show. “We would like to express our warmest thanks to the patients, carers and investigators for participating in the REFALS…