The National ALS Registry and Biorepository was created back in 2007 to help understand how prevalent ALS is, who is developing ALS, and what the possible causes are. Its mission is also to help support researchers in discovering treatments and cures and in preventing ALS.
After Amylyx’s announced that it intends to file a new drug application for AMX0035, The ALS Association immediately called on the U.S. Food and Drug Administration to approve the treatment for all people with ALS as soon as possible. Connecting ALS talked to the team at Amylyx to learn about the path ahead for access to AMX0035.
There is a lot to do, and this grounded focus of making ALS livable helps us hold everyone—ourselves, the FDA, and the research community—accountable to real impacts on real people with ALS and the time it takes to deliver those impacts. This week has been a big step forward for the ALS community, and we will continue urgently working to keep the momentum going.
We recently talked with Dr. Yichen Li, postdoctoral fellow from the Ichida Lab at the University of Southern California to learn about her unique project focused on the efficacy of suppressing a gene called SYF2 as a therapeutic strategy for diverse forms of ALS.
Dr. Jeffrey Rothstein, professor of neurology and neuroscience and the founding director of the Robert Packard Center for ALS Research at Johns Hopkins University School of Medicine, and Dr. Alyssa Coyne, a postdoctoral fellow at Johns Hopkins, discuss their recent publication of research identifying a cellular defect common in ALS and what it means for research into the disease going forward.
We recently spoke with Nishal to learn more about him and his unique project focused on providing an assistive communication device for people with severe speech and motor impairment due to ALS using an intracortical Brain Computer Interface (iBCI).
In very powerful testimony, members of the ALS community asked members of Congress to ensure the FDA acts with greater speed and regulatory flexibility during a hearing before the House Energy and Commerce Health Subcommittee.
As part of this year’s National Advocacy Conference, Dr. Bryan Traynor, senior investigator and chief of neuromuscular diseases at the NIH’s National Institute on Aging presented a case study to the ALS community to help better understand what we know today and what we are working toward for the future relating to genetics and prevention. ALS prevention has not only been a priority for Traynor, but for the ALS community at large.
Dr. Lauren Gittings, whose award was made possible directly through funds provided by The ALS Association Oregon and SW Washington Chapter, is a postdoctoral fellow from the Sattler Lab at the Barrow Neurological Institute in Phoenix, Arizona. We recently spoke with Lauren to learn more about her and her unique project focused on identifying cellular and molecular changes that underlie cognitive impairment in ALS patients carrying the C9orf72 (C9) repeat expansion mutation.
We recently talked with Dr. Sonia Vazquez-Sanchez, postdoctoral fellow from the Cleveland Lab at the Ludwig Institute for Cancer Research at the University of California at San Diego to learn about her unique research focused on RNA binding protein TDP-43 and its effects on ALS.
Following our We Can’t Wait Action meeting with the FDA, we sent a letter to the agency asking it to reaffirm its commitment to the 2019 ALS Guidance and provide us with a detailed report showing how it has implemented the guidance. This week, the ALS Association received a response from Dr. Patrizia Cavazzoni, Director of the Center for Drug Evaluation and Research.
Investigators at Emory University School of Medicine reviewed 23 years of data from 1997-2020 for patients seen at the Emory ALS Center. To allow for adequate analysis of disease survival time, researchers included all patients who self-reported their race as Black or White and symptom onset was before January 1, 2017. A total of 1,298 patients were included in the study, 203 of whom were Black, and 1,095 of whom were White.
People with ALS on Tuesday called on the FDA to use its existing authority and the flexibility it promised the ALS community it would use to make experimental drugs that show incremental benefits available as quickly as possible. The comments came in a “We Can’t Wait” Action Meeting with FDA organized by The ALS Association.
We are deeply grateful to the eight speakers who shared their wisdom and personal experience with the FDA, as well as the more than 150 others from the community who have done so online. We continue to hear that people with ALS want effective treatments now, even if they offer modest benefit, and are willing to face considerable uncertainty and risk to do so.
Later this month, the ALS Association will host a virtual session focused on the urgent need for timely access to new therapies. Leaders from the U.S. Food and Drug Administration (FDA) and industry companies will listen to people living with ALS speak directly about their expectations for their experiences with the disease and how important it is to have timely access to therapies that have the potential to provide incremental benefit in improving how people feel, function and live.
Dr. Jan Veldink is receiving the award in recognition of his contribution to ALS epidemiology and genetics which have led to important novel discoveries including several new ALS risk genes. He has established a research line on ALS genetics, epidemiology and transcriptomics and has a proven track record in both array-based and sequencing technology. In addition, he has established an international biobanking register and patient database specifically for ALS, which is essential to be able to carry out Project MinE.
The ALS Association, in partnership with The CReATe Consortium, is proud to announce a new request for applications (RFA) to support the discovery and/or validation of biomarkers for ALS, a third grant funding opportunity now available to researchers seeking to find new treatments and a cure.