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…
A French national agency has cleared AB Science to resume patient enrollment in the Phase 3 clinical trial investigating masitinib in people with amyotrophic lateral sclerosis (ALS), the company announced in a press release. AB had voluntarily suspended recruitment and treatment in all its masitinib clinical studies in…
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…
I inspected the fish through the curved glass of a refrigerated display case. Wild-caught Lake Superior whitefish and trout — both raw and smoked — lay in metal trays. “I’d like that piece of smoked trout in the middle. Underneath the top one,” I specified. I picked a plump…
A Phase 1b clinical trial evaluating the safety and pharmacological profile of DNL343, Denali Therapeutics’ investigational oral therapy, in adults with amyotrophic lateral sclerosis (ALS) has started dosing participants. The study (NCT05006352) is currently recruiting patients at the Centre for Human Drug Research (CHDR) in Leiden, Netherlands.
“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…
Even though I believe I’m a pretty good planner and can strategize with the best of ‘em, last week threw me into a planning frenzy. I was anticipating my first in-person ALS clinic visit after nearly a year of online appointments with my neurologist. Pre-pandemic, I had many in-person…
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