News

Specific alterations in RNA processing are molecular hallmarks of familial and sporadic forms of amyotrophic lateral sclerosis (ALS), a new study suggests. This research might lead to more discoveries  about how ALS develops and ultimately contribute to a cure. The study, “Intron retention and nuclear loss of SFPQ are…

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…

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…

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…

AB Science has decided not to seek the re-examination it initially requested after the European Medicines Agency (EMA) issued a negative opinion on marketing authorization for masitinib, the company’s investigational amyotrophic lateral sclerosis (ALS) therapy. The company announced in a press release that the re-examination procedure…