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Top ALS News Today News
Norway · NorwayTaking statins to lower cholesterol does not affect survival outcomes for people with amyotrophic lateral sclerosis (ALS), researchers in Norway have found. In a study of ALS patients using national health registry data, the scientists compared the life expectancy of statin users and nonusers. According to the team, “statin use was not associated with ALS survival.” The researchers noted that many people stop using statins prior to receiving an …See the Story
In ALS, statin use not related to patient survival, study finds
Maryland · MarylandNine patients with an ultra-rare form of amyotrophic lateral sclerosis (ALS) caused by mutations in the CHCHD10 gene were treated with an experimental antisense oligonucleotide (ASO) that’s designed to silence the disease-causing mutations. The treatment was developed as part of Silence ALS, an initiative that designs ASOs, that is, short strands of genetic material, for carriers of ultra-rare, disease-causing genetic mutations. The effort was b…See the Story
Silence ALS treats 9 patients' ultra-rare ALS caused by CHCHD10...
United States · United StatesThe U.S. Food and Drug Administration (FDA) has authorized Sineugene Therapeutics to begin a Phase 1/2a trial of its experimental gene therapy, SNUG01, in people with amyotrophic lateral sclerosis (ALS). The global trial will assess the treatment’s safety, tolerability, and preliminary efficacy through a dose escalation and expansion protocol in adults with ALS, according to a company press release. ALS is caused by the progressive dysfunction a…See the Story
FDA approves study of SNUG01 gene therapy for adults with ALS
Baltimore, MD · BaltimoreThe weeks immediately following my late husband’s ALS diagnosis were the scariest and most unsettling of my life. Even with Jeff’s physical symptoms pointing toward ALS, nothing could’ve prepared us to actually hear those words when they were delivered in a windowless examination room in Baltimore, in November 2018. In many respects, those initial days of shock and despair were more difficult than any that followed. Jeff and I each retreated in …See the Story
Sharing fears with other ALS caregivers can help us feel less alone
Australia · AustraliaNUZ-001, which was previously known as monepantel, slowed lung function declines by nearly 50% in people with amyotrophic lateral sclerosis (ALS), according to new analyses from a Phase 1 trial. This slowing of respiratory declines was strongly correlated with slower declines in overall function that were observed in previous analyses. Developer Neurizon Therapeutics is using the data to finalize its plans for NUZ-001 to enter the HEALEY ALS pla…See the Story
NUZ-001 shows positive early results, headed toward HEALEY trial
Seville · SevilleCertain subsets of natural killer (NK) cells, a part of the immune system, were elevated in the blood of people with amyotrophic lateral sclerosis (ALS), where they showed altered gene activity and signaling patterns, a study found. One subset was associated with altered immune signaling, while another was linked to neurodegeneration. Other immune cell types also showed changes suggestive of an overall dysregulated peripheral immune system — the…See the Story
Study highlights role of natural killer immune cell subsets in ALS
United States · United StatesIt’s hard to live in limbo with the slow but relentless progression of my husband Todd’s ALS. A couple years after Todd was diagnosed, he went on permanent disability from his work. He could still speak and had the time, so he visited my great-uncle nearly every day in a long-term care facility to read to him. Halfway through Herman Melville’s “Moby-Dick,” Uncle Carl passed away. Todd tried to plow through the rest of the whaling chapters but co…See the Story
How we’re living in the middle chapters of ALS
Mississippi · MississippiScientists have developed a novel method to efficiently grow motor neurons from skin cells, which may serve as a platform for creating cell therapies to treat diseases like amyotrophic lateral sclerosis (ALS). The new method was described in two papers — one titled “Compact transcription factor cassettes generate functional, engraftable motor neurons by direct conversion” and the other “Proliferation history and transcription factor levels drive…See the Story
