Nine 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,…
Silence ALS treats 9 patients’ rare ALS caused by CHCHD10 mutations
The 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…
People living in California can help fund amyotrophic lateral sclerosis (ALS) research when filing their state taxes. The California ALS Research Network Voluntary Tax Contribution Fund allows Californians to contribute to research projects selected by the ALS Network’s scientific advisory committee. At least $250,000 must be raised…
The 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.
NUZ-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…
It’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…
Certain 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…
Scientists 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…
If you’ve been keeping up with my recent columns, you’ve likely figured out that even though I have ALS, I maintain a glass-half-full attitude. So, today, I thought you’d enjoy learning how I deal with what has been a constant challenge for me: plastic bottles. The trouble with plastic…
Health Canada has invited Neurosense Therapeutics to discuss the potential for giving conditional approval to PrimeC in treating amyotrophic lateral sclerosis (ALS), according to a company filing with the U.S. Securities and Exchange Commission. The upcoming meeting provides an opportunity to consider if the drug is eligible…
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