A Phase 1/2 clinical trial investigating ION541, Ionis Pharmaceuticals‘ third antisense treatment and its first for sporadic, rather than familial, amyotrophic lateral sclerosis (ALS), has started dosing, the company announced. This ascending dose safety trial (NCT04494256) is recruiting up to 70 patients…
Dosing Begins in Trial of ION541, Antisense Treatment for Sporadic ALS
People with diabetes mellitus, particularly those with type 2 diabetes, appear to be at a significantly lower risk of amyotrophic lateral sclerosis (ALS) than those without this metabolic disorder, a review study reported. Its findings support previous research suggesting diabetes to be a protective factor against ALS, potentially…
Catalent Biologics has agreed to manufacture NurOwn, the cell-based therapy by BrainStorm Cell Therapeutics being evaluated in a soon-to-conclude pivotal trial as a possible treatment of amyotrophic lateral sclerosis (ALS). With this agreement, Catalent will produce NurOwn under current Good Manufacturing Practices — standards set to ensure that batches…
A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like the option of continuing its use in future appointments, a series of surveys found. The surveys were conducted by the National Organization for Rare Disorders (NORD) and involved more than 800…
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We Are Both Patients
I asked my husband, Todd, why he thought our relationship worked as well as it does. I was preparing to write a column about how we still love each other through the stress of living with his ALS for a decade now. It would be understandable for him to…
MND-SMART, a pivotal platform trial designed to simultaneously test multiple treatment candidates in people with amyotrophic lateral sclerosis (ALS) and other motor neuron diseases, has opened its third clinical site in Scotland. This type of platform trial, distinct from typical clinical trials that test a single treatment…
“Won’t you look down upon me, Jesus?/ You gotta help me make a stand./ You just got to see me through another day./ My body is achin’ and my time is at hand./ I won’t make it any other way.”…
A $2 million grant from the National Institutes of Health (NIH) is funding work to better understand how molecular structures called protein-RNA condensates form and are regulated within cells. Findings from the five-year project could help in understanding and treating diseases where these molecular structures play a role, including…
It’s always gratifying to know what I write is helpful to others living with ALS. Recently, I wrote about how I periodically take the time to review my daily habits and set new goals for the month. While readers left many comments thanking me, others asked if I had…
Head trauma, electrical burns, and hobbies involving the toxic metal lead appear to be risk factors for developing amyotrophic lateral sclerosis (ALS), a U.S.-based study found. Holding a job in mechanics, painting, or construction also significantly increase the chances of developing the disease, researchers noted. The study, “…
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