Flex Pharma announced that it is stopping Phase 2 clinical trials of its investigational compound to ease muscle cramps, FLX-787, in patients with amyotrophic lateral sclerosis (ALS) and Charcot-Marie-Tooth disease (CMT). The company’s decision was based on tolerability concerns in each study with the oral disintegrating tablet formulation at 30…
Researchers have developed a personalized, non-invasive model that uses a set of characteristics, easily obtained at diagnosis, to predict survival in patients with amyotrophic lateral sclerosis (ALS). The study with that finding, “Prognosis for patients with amyotrophic lateral sclerosis: development and validation of a personalised prediction model,” was published…
Patients with amyotrophic lateral sclerosis (ALS) are at increased risk for having pressure sores (PrS), according to a recent study. This risk is especially high for women and young patients, the findings showed. The study “Risk of developing PrS in amyotrophic lateral sclerosis patients – a nationwide cohort…
Cognitive deficits associated with amyotrophic lateral sclerosis (ALS) are linked to a particular pattern of abnormal changes and altered blood flow in specific brain regions, a study shows. The study titled “Brain Structural and Perfusion Signature of Amyotrophic Lateral Sclerosis With Varying Levels of Cognitive Deficit” was…
The biotech company Kadimastem is recruiting for a Phase 1/2a clinical trial to test the safety and effectiveness of its leading cell therapy, called AstroRx, in patients with amyotrophic lateral sclerosis (ALS), after positive results were seen in animal models of the disease. The study showed that AstroRx, made…
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…
“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…
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