An ambitious Irish research project, called Precision ALS, will combine clinical research, data science, and artificial intelligence (AI) to identify the multiple — and potentially targetable — factors involved in the development and progression of amyotrophic lateral sclerosis (ALS). The goal is to use this information to determine which treatments…
News
Exposure to certain toxic compounds — including beta-N-methylamino-L-alanine (BMAA), formaldehyde, and heavy metals like manganese, mercury, and zinc — increases the likelihood of developing amyotrophic lateral sclerosis (ALS), according to a review paper. Its authors argue that with BMAA — a toxic compound made by algae — enough evidence…
Patient registries are a hot topic of rare disease research and many organizations are taking advantage of this resource by signing up their patient communities and connecting with researchers. Eric Sid, MD, program officer for the Office of Rare Diseases Research (ORDR), said it is difficult to estimate how…
University of Sydney neurologist Matthew Kiernan, PhD, has been granted the 2022 Sheila Essey Award for his groundbreaking research, aimed at providing earlier diagnoses and better treatments for people with amyotrophic lateral sclerosis (ALS). Kiernan, a world-leading expert in the field of ALS research, is the first Australian to…
A Phase 1 clinical trial investigating ProJenX‘s oral therapy prosetin in healthy volunteers and people with amyotrophic lateral sclerosis (ALS) has started dosing participants. The first-in-human PRO-101 trial is divided into three parts. In parts 1a and 1b, researchers will investigate the safety, tolerability, and pharmacokinetics of single and…
Haisco Pharmaceutical has acquired the exclusive rights to develop and commercialize Exservan, an oral film formulation of riluzole, for the treatment of amyotrophic lateral sclerosis (ALS) in China. Under the terms of the agreement, Haisco will be responsible for the potential regulatory submission and marketing of Exservan in China, while…
The ALS Association has formally objected to the use of two controversial measures of cost-effectiveness in the upcoming evaluations of AMX0035 and oral edaravone, which are being reviewed as treatments for amyotrophic lateral sclerosis (ALS) in the U.S. These cost-effectiveness measures fail to value the lives of ALS…
A two-year project aims to identify reliable blood tests for measuring neurofilament protein levels, which could aid in the early detection of frontotemporal degeneration (FTD) and amyotrophic lateral sclerosis (ALS), and possibly other rare neurodegenerative disorders. Neurofilament, a protein component of neurons, is released when these nerve cells are…
The Phase 2/3 clinical trial testing UCB’s investigational therapy zilucoplan for amyotrophic lateral sclerosis (ALS) — one of the arms of the multi-regimen HEALEY ALS platform trial — has been stopped early based on interim data. The decision was made after a pre-specified analysis “demonstrated futility,” UCB said…
Horizon Therapeutics has launched its #RAREis Representation program aimed at increasing diversity, equity, and inclusion among patients with rare diseases. There are about 400 million people worldwide living with a rare disease; for many of them, access to diagnosis, care, and treatments can be challenging. Accessing better care depends on…
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