BL-001 demonstrated a favorable safety and tolerability profile in healthy volunteers. These results indicate that BL-001 can be given safely to humans at doses up to 10 times more than what was needed for efficacy in animal models. Bloom Science received funding from The ALS Association’s Lawrence and Isabel Barnett Drug Development Program to advance BL-001’s development.
The non-profits have awarded seven new grants worth a total of $2 million over the next 3 years to support the development of early diagnostics for ALS.
Mutations in the NEK1 gene, which was discovered as a direct result of Ice Bucket Challenge funding, were found to destabilize the structure of neurons and disrupt their ability to import cellular cargo into the nucleus.
New results from the phase 3 Study 19/MCI186-19 suggest that people with ALS who received IV Radicava first and continued with treatment through the open-label extension had a lower risk of death, tracheostomy, permanent assisted ventilation or hospitalization compared to those who received placebo first followed by Radicava.
ALS Focus participants told us back in 2020 that mobility and being able to do activities matter most to people living with ALS. We want to know more so we can more effectively address the needs of our community. From now until October 23, 2023, people with ALS, current caregivers and past caregivers are encouraged to participate in the ALS Focus Mobility at Home Survey. The
information and insight we collect will help us measure access to, costs of and quality of this equipment so we can advocate for stronger insurance coverage and better overall care. For more information, visit www.ALSFocus.org or email ALSFocus@als.org.
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 U.S. 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, our programs and awards, and the projects we fund, 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@als.org.