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ALS Association
Reasons for Hope in ALS Research with the Discovery of NU-9
In a study funded in part by The ALS Association’s TREAT ALS program, researchers from Northwestern University have identified the first compound (NU-9) that eliminates the ongoing degeneration of diseased upper motor neurons, a key contributor to ALS. While this news is exciting, this study has only tested the compound in mice and in laboratory neurons and is in the very early stages.
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ALS Association
What ALS Focus Hopes to Learn from ALS Caregivers
ALS Focus recently launched its third survey, gathering data from ALS caregivers to identify the unique needs and challenges of ALS caregiving. Responses to this survey will help translate the caregiver experience into action and influence ALS programs and policy decisions. ALS Focus Director Dr. Sarah Parvanta detailed the questions her team is asking in the current survey during a recent episode of Connecting ALS. A portion of that conversation has been edited and condensed below.
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ALS Association
Biogen Shares Latest Updates on ALS Clinical Trials
Despite the many challenges caused by COVID-19 this past year, ALS research made strides toward finding effective treatments for ALS. As 2020 winds down, we wanted to share an update from our partners at Biogen on some of their ongoing research projects.
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ALS Association
AMX0035 Petition Delivered to FDA
The ALS Association and I AM ALS on Friday submitted a petition to the Food and Drug Administration calling on the agency and Amylyx Pharmaceuticals to act swiftly and with urgency to make AMX0035 available as soon as possible. The petition was signed by more than 50,000 people from across the country who have been affected by ALS.  
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ALS Association
AMX0035 Survivability Data Adds to Urgency to Make Drug Available
Clinical trial participants who took AMX0035, a promising new drug therapy developed by Amylyx, showed a statistically significant 6.5 month increase in survivability compared to patients who did not receive the drug in the initial trial, according to data published in the journal Muscle and Nerve in October 2020. These findings validate calls led by The ALS Association and I AM ALS for Amylyx and the FDA to make AMX0035 available as quickly as possible. 
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ALS Association
Jinsy Andrews: ALS Researcher in Search of a Cure
ALS doesn’t stop and neither do we. The reality is, people living with ALS can’t wait for treatments and a cure, and just as importantly, the tireless researchers working together around the world can’t wait to make the next breakthrough. 

Meet Jinsy Andrews, M.D., MSc, FAAN, Director of Neuromuscular Clinical Trials at Columbia University, and member of The ALS Association Board of Trustees. Dr. Andrews is a clinical neurologist, neuromuscular specialist, and an ALS specialist. 
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ALS Association
Researcher Spotlight: Emily Thompson, Ph.D.
We recently talked with Dr. Emily Thompson from the Rothstein Lab at Johns Hopkins University to learn about her unique research project focused on how the loss of a cortical astroglia subpopulation exacerbates dendritic and synaptic defects of upper motor neurons in ALS.
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ALS Association
Researcher Spotlight: Valeria Gerbino, Ph.D.
We recently talked with Dr. Gerbino from the Maniatis Lab at Columbia University to learn about her unique research project focused on identifying how mutations in TBK1, one of the genes associated with ALS, differentially affect the cells of the spinal cord involved in the pathogenesis of ALS.
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ALS Association
Researcher Spotlight: Paul McKeever, Ph.D.
We recently spoke with Dr. Paul McKeever from the Rogaeva lab at the Tanz Centre for Research in Neurodegenerative Diseases at the University of Toronto. Paul’s current research project is focused on uncovering the molecular programming which make individual brain cells and populations of cells susceptible or resilient to the disease process so that new therapeutic avenues can be developed for patients with ALS and FTD.
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ALS Association
Researcher Spotlight: Lauren Laboissonniere, Ph.D.
We recently talked with Dr. Lauren Laboissonniere from the Ranum lab at the University of Florida to learn about her unique research project focused on the development of novel therapeutics for the treatment of C9orf72 ALS/FTD and related repeat-associated disorders.
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ALS Association
Biogen Announces Latest Updates on ALS Clinical Trials During the Pandemic
Biogen, a partner of The ALS Association, recently published promising results from its phase 1–2 Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS and is now actively enrolling participants for their Phase 3 Valor study. It also announced that there is an open-label extension available in the study.
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