It will take all of us, working together, to urgently propel research to make ALS a livable disease until new treatments and a cure are ultimately found.
This month, learn more about the latest news and updates on Amylyx and AMX0035, promising results from the Tofersen trial and the importance of investing in assistive technology to help empower people living with ALS to live life on their own terms and help reduce harms associated with the disease.
The ALS Association recently joined other ALS organizations releasing an open letter urging the FDA to quickly approve AMX0035, especially given the recent approval of the drug by Health Canada. The FDA was originally scheduled to reach a decision on approval in the US no later than June 29 but extended the time period by three months to consider additional data.
Follow-up analyses from the Phase 3 Valor study and the open-label extension study of tofersen, an investigational antisense drug, suggested that the drug was effective in slowing down progression of ALS in people with SOD1 mutations.
Amylyx Pharmaceuticals announced that Health Products and Food Branch (HPFB) of Health Canada officially approved ALBRIOZA, known as AMX0035 in the US, a new treatment for ALS. Clinical trials showed the drug to be safe and effective for people living with the disease, and the FDA is currently considering whether to approve the drug for patients in the US.
ICER (the Institute of Clinical and Economic Review) issued its initial draft report on the cost-effectiveness of AMX0035 and oral edaravone. The organization purports to assess whether a drug has value so payers (health insurance companies) can make decisions about whether to provide coverage and fair pricing for the drug. The methodology ICER uses is discriminatory to seniors, people with chronic illnesses and disabilities.
A wide range of equipment and technologies can help improve mobility and manage symptoms as the disease progresses making it easier for people living with ALS to get around their homes, speak and perform day-to-day tasks. Supporting scientific advancements in assistive technology is just one focus area enabling us to help improve the lives of people living with ALS.
ALS Focus allows people living with ALS, and current and former caregivers the ability to participate in research outside of clinical trials and is available to all at any stage of the disease. Your opinions matter to help strengthen care, accelerate therapy development, improve clinical trials, influence insurance coverage decisions, and more.
Register to participate in future surveys HERE and let your voice be heard.
Dr. Stanley Appel, a world-renowned expert in ALS and the founder of the first multidisciplinary clinic dedicated to ALS care and research in the US, received the MDA Tribute Award acknowledging his incredible work in pioneering ALS research and care at Houston Methodist Neurological Institute's MDA ALS Research and Clinical Center, an Association Certified Center of Excellence.
Clinical research is the only way that promising laboratory science can be translated into treatments and cures for ALS and other neurological diseases. People with ALS, along with family members and caregivers, are essential partners in this research. For the most accurate and up-to-date information on both federally and privately funded clinical studies focusing on ALS and other similar motor neuron diseases, visit our website.
The National ALS Registry may be the single largest ALS research project ever created and is designed to identify ALS cases from throughout the entire United States. Every person living with ALS in the US can take part and there is no fee to participate.
The ALS Association partners with the Northeast ALS Consortium (NEALS) to provide educational webinars for people with ALS, caregivers, and the broader ALS community. Topics include updates on clinical trials, best practices for clinical care, and current research efforts in ALS causes and treatments.
For more detail about current ALS research, the projects we fund, and the numerous focus areas we support, visit als.org/research.
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We welcome all feedback, comments and questions you may have on anything about the information provided, suggestions of topics you would like to see in future newsletters or thoughts to help us improve. Please email us at communication@alsa-national.org.