- Amyotrophic Lateral Sclerosis Insights
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- Amyotrophic Lateral Sclerosis Insights
Amyotrophic Lateral Sclerosis Insights
This week's must-know community updates, latest research & events
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Apply now: feature your voice in our cell & gene therapy series Cell and gene therapies are transforming rare disease treatment, offering new hope where options were once limited. But how do they work, and what choices exist for your community? We're launching a special content series to help our 15k+ readers navigate this evolving landscape – and we need your voice! Be part of the conversation, whether you are: • A patient or carer • A patient support group • Developing cell & gene therapies • Administering cell & gene therapies 📅 Enquiries close: March 12 Let's educate, advocate and drive change together. | ||||||||||||||||||||
Top StoriesWoolsey Pharmaceuticals Advances ALS Treatment Funding Funding will help Woolsey Pharmaceuticals continue developing BRAVYL, a potential treatment for amyotrophic lateral sclerosis, which has shown positive results in reducing certain ALS markers. Read More →NfL Biomarker Improves ALS Diagnosis and Prognosis Measuring blood levels of NfL helps diagnose ALS and predict survival by indicating nerve damage. This biomarker is more accurate than others for ALS, aiding in disease progression tracking and treatment evaluation. Read More → | ||||||||||||||||||||
Latest ResearchIn a study by Eijk et al. (2025), the safety and effects of trimetazidine on patients with amyotrophic lateral sclerosis (ALS) were assessed. The medication, known to inhibit partial fatty acid oxidation, was given to 21 participants over a 12-week period. The study primarily looked at how well patients tolerated the drug and its impact on oxidative stress markers, which are indicators of cell damage. Results showed that 90% of participants completed the treatment, and trimetazidine was generally well-tolerated, with only 11% of adverse events possibly related to the drug. Notably, there was a significant reduction in the oxidative stress marker 8-OHdG during treatment, suggesting less cellular damage. Additionally, patients experienced a decrease in resting energy expenditure, which could indicate improved metabolic efficiency. These promising findings support the need for a larger, more controlled study to further explore trimetazidine's potential benefits for ALS patients. | ||||||||||||||||||||
Community News
Health Canada has approved QALSODY (tofersen injection) under a Notice of Compliance with Conditions (NOC/c) for the treatment of ALS in adults who have a pathogenic variant in the SOD1 gene. This means the drug will soon be made available and can be marketed in Canada with certain conditions.
We are excited to welcome Allison Bulat, Chief Engagement Officer, to the ALS Hope Foundation team!! 💙💛 | ||||||||||||||||||||
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