Results from a Phase 1 clinical trial reveal that giving patients infusions of a specialized immune cell may be a viable option to safely slow the progression of amyotrophic lateral sclerosis (ALS). That finding was reported in the study “Expanded autologous regulatory T-lymphocyte infusions in ALS,” published…
Canada’s healthcare system is excellent for people with common ailments like diabetes or high blood pressure, but it’s “basically failing the nearly three million Canadians with rare diseases.” So says Durhane Wong-Rieger, president and CEO of the Canadian Organization for Rare Disorders (CORD), a Toronto-based network representing 102 patient advocacy…
“I’m afraid you have amyotrophic lateral sclerosis.” My neurologist continued, “Anyone can get it, even myself,” and like Charlie Brown in the classroom, I shut him off completely, reducing his vocal output to a series of horn mutes. I then went home and googled ALS. Devastated by what I…
A high genetic risk for amyotrophic lateral sclerosis (ALS), assessed using so-called polygenic risk scores, was linked in a study to poorer performances on verbal and numerical tests in otherwise healthy adults, but not to physical disabilities. The study, “Genetic risk for neurodegenerative disorders, and its overlap with cognitive…
Just as no two ALS journeys are alike, there is no one-size-fits-all method for dealing with the emotional stress of living with this disease. During this past year of columns, I’ve shared many of the tips and strategies that I use to maintain a positive mindset…
A new portable system provides insights into the altered tongue, lip, and jaw motion in amyotrohic lateral sclerosis (ALS) patients with a speech disorder called dysarthria. The instrument could be valuable for both diagnosis and treatment of speech impairment. The study, “Articulatory Range of Movement…
As President Trump signed the recently passed Right to Try legislation into law in a White House ceremony, Jordan McLinn of Indianapolis tried twice to embrace him. The third time, 9-year-old Jordan finally got the hug he wanted — as well as a kiss on the forehead. The video of…
Lower-than-usual levels of a molecule called miR126-5p — a type of microRNA, with a role in producing proteins — led to neuron degeneration and disease progression in a mouse model of amyotrophic lateral sclerosis (ALS), in part by working to raise levels of toxic molecules, a study reports. Its researchers…
Two enzymes called USP5 and USP13 were found to have an important role in fighting back against toxic “stress granules,” a study reports. The study, titled “Deubiquitylases USP5 and USP13 are recruited to and regulate heat-induced stress granules through their deubiquitylating activities,” and published in the …
An experimental oral formulation of edavorone for amyotrophic lateral sclerosis (ALS), called TW001, demonstrated promising pharmacological and safety data in a Phase 1 clinical trial, according to the biotech Treeway. The randomized trial compared TW001 to Radicava (Mitsubishi Tanabe Pharma America), an intravenous medication approved…
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