The United Kingdom-based Motor Neurone Disease (MND) Association was commended recently at the Third Sector Awards for its efforts in the MND community. The nonprofit organization won high commendations in the “Charity of the Year: Over £5M” category at the annual awards, which recognizes the achievements of charitable organizations and…

QurAlis is advancing the development of QRL-201, its “groundbreaking” lead treatment candidate for amyotrophic lateral sclerosis (ALS), and expects to initiate its first clinical trial in late 2022. The stem cell technology company said it is now conducting formal preclinical studies to assess QRL-201’s therapeutic potential, and expects…

The two most common measures used in amyotrophic lateral sclerosis (ALS) assessments — the ALS functional rating scale-revised (ALSFRS-R) and forced vital capacity (FVC) — can predict survival more accurately when used together, according to a recent study. Alone, these measures had a similar predictive ability, but the weak…

“Just what makes that little old ant Think he’ll move that rubber tree plant Anyone knows an ant can’t Move a rubber tree plant But he’s got high hopes …” — Frank Sinatra, “High Hopes” Recently, I watched the movie “A Hole in the Head” for the first time…

Amylyx Pharmaceuticals plans to submit a new drug application (NDA) to the U.S. Food and Drug Administration (FDA) in the coming months for approval of its investigational oral therapy AMX0035 for amyotrophic lateral sclerosis (ALS). The decision follows recent discussions with the FDA, including a pre-NDA meeting held…

A newly launched non-profit institute is seeking to advance research, and the development of new therapies, for people with rare diseases — a patient community with some of the largest therapeutic needs, but one that is often left behind. Named the Institute for Life Changing Medicines, the project was…

Note: This story was updated Sept. 21, 2021, to note that 18% of patients, not 80%, experienced increases in the MUNIX(4) sum score at some point during the study. The last patient visit has been completed in Clene’s Phase 2 trial investigating the potential of CNM-Au8 — which…

Participation in clinical trials exposes rare disease patients to financial, physical, and emotional pressures, according to the results of a patient focus group series. “Rare disease trial participants are running an endurance race they are highly motivated to complete, but these incremental burdens negatively impact their ability or willingness to…

I watched a tribute video to a husband and father who recently died from ALS. Tears filled my eyes over tender moments of a toddler kissing and hugging his dad, jumping on his lap, and driving matchbox cars on his head while the dad smiled. The man radiated love for…

A $10-million grant from the National Institutes of Health (NIH) will help fund the first U.S.-based clinical trial of Synchron’s Stentrode, a wireless brain device, implanted without open brain surgery, that transmits signals from the brain directly to a computer. The COMMAND trial will evaluate whether the device…