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 tofersen, AMX0035 and oral edaravone, HEALEY Platform Trials and the importance of investing in the study of ALS disease models to help identify new treatments for the disease.
The FDA has accepted Biogen’s new drug application for tofersen, a potential treatment for people with SOD1 mutations that cause ALS. The FDA granted priority review and will make a decision about whether to grant accelerated approval for tofersen by January 25, 2023.
The ALS Association filed formal objections with the Institute for Clinical and Economic Review, commonly known as ICER, over their flawed draft report on the cost-effectiveness of AMX0035 and oral edaravone.
As scientific researchers continue to uncover new genetic links to ALS, the rise of gene therapies to treat the disease will remain a development to watch in the coming years. Kuldip Dave, Ph.D., senior vice president of research at The ALS Association shares the progress being made and the promising future it has in discovering new treatments and cures for ALS.
The Healey & AMG Center for ALS at Mass General and Calico Life Sciences LLC recently signed an agreement to work together to test ABBV-CLS-7262 in the HEALEY ALS Platform Trial.
Clene Nanomedicine reported significantly improved survival in ALS patients initially treated with CNM-Au8 compared to initially randomized placebo treated participants during the long-term open-label extension of its RESCUE-ALS trial.
ALS disease models used in the laboratory help researchers understand the basic processes of the disease, which is essential for developing new therapies. Many are used to test new therapies to identify those compounds or groups of compounds that are promising enough to bring to clinical trial in 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. Ahmad Al Khleifat, recent winner of the distinguished ENCALS Young Investigator Award and postdoctoral fellow from King’s College London, discusses his research focused on disease gene identification through next generation sequencing, coupled with advanced data analysis to deliver diagnostic tools for complex disease genetics.
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.