Target ALS

Annie Ratliff Brandt is running the 2024 NYC Marathon with a deeply personal connection to ALS. Four years ago, she lost her father, but his best friend Scott, diagnosed with ALS 15 years ago, continues to inspire her. Despite ALS, her father and Scott never missed their annual hunting and camping trips, finding creative ways to keep their adventures alive. Whether it was her father carrying Scott up a hill or Scott bringing his cane, their friendship and resilience never wavered. When her father became ill, Annie, newly engaged, was unsure who would walk her down the aisle. Scott, in a show of friendship and love, immediately offered to step in if needed. Thankfully, her father was able to walk her down the aisle before he passed away a few months later. Throughout that difficult time, Scott was by Annie and her family's side, sharing stories and memories of her father, offering support, and reminding her of her father’s enduring love. “To honor my dad and my dad’s best friend Scott, I am honored and thrilled to be running the New York City Marathon for Target ALS.” Annie is running to support Target ALS’ mission to break down barriers to ALS research to find effective treatments to realize a world where #EveryoneLives Donate today: https://bit.ly/3BsqXba #NYCMarathon #ALSResearch #Fundraiser #EveryoneLives

Annie Ratliff Brandt is running the 2024 NYC Marathon with a deeply personal connection to ALS. Four years ago, she lost her father, but his best friend Scott, diagnosed with ALS 15 years ago, continues to inspire her. Despite ALS, her father and Scott never missed their annual hunting and camping trips, finding creative ways to keep their adventures alive. Whether it was her father carrying Scott up a hill or Scott bringing his cane, their friendship and resilience never wavered. When her father became ill, Annie, newly engaged, was unsure who would walk her down the aisle. Scott, in a show of friendship and love, immediately offered to step in if needed. Thankfully, her father was able to walk her down the aisle before he passed away a few months later. Throughout that difficult time, Scott was by Annie and her family's side, sharing stories and memories of her father, offering support, and reminding her of her father’s enduring love. “To honor my dad and my dad’s best friend Scott, I am honored and thrilled to be running the New York City Marathon for Target ALS.” Annie is running to support Target ALS’ mission to break down barriers to ALS research to find effective treatments to realize a world where #EveryoneLives Donate today: https://bit.ly/3BsqXba #NYCMarathon #ALSResearch #Fundraiser #EveryoneLives

Meet Kathryn Morelli, Ph.D., an academic investigator and Assistant Professor at University of Vermont, who received a Springboard Fellowship from Target ALS in partnership with ALS United Greater New York. Dr. Morelli’s research is advancing zinc finger nuclease technology, a gene-editing tool, for potential treatment of c9orf72 ALS. The Springboard Fellowship program supported the transition from her postdoctoral fellowship to her faculty appointment at University of Vermont, leading cutting-edge ALS research. Through Springboard Fellowships, Target ALS aims to support talented scientists at early stages in their careers to foster their interest and encourage new ideas on ALS research, paving the way for tomorrow’s breakthroughs. Thank you to ALS Greater United New York for joining us in supporting Dr. Morelli’s exciting work, exemplifying the radical collaboration needed to accelerate the discovery of new drugs and biomarkers. We are creating a dynamic environment where scientific innovation can thrive, driving us closer to our shared goal: building a future where #EveryoneLives. Read more on the Target ALS Springboard Fellowship here: https://bit.ly/3BAvk41 #RadicalCollaboration #ALSFellowship #TargetALS #ALSResearch #NonprofitPartnerships #BreakingBarriers #ALSUnited

We are thrilled to share that Instituto Roosevelt in Bogotá, Colombia, has enrolled 5 participants within the last month in the Target ALS Global Natural History Study. This milestone reflects our commitment to accelerating ALS research through global collaboration and the inclusion of often underrepresented ethnicities. By emphasizing genetic diversity, we are unlocking deeper insights into the disease's biology and paving the way for targeted therapies that can benefit everyone living with ALS. This study plays a pivotal role in our vision of building a future where #EveryoneLives Learn more about the Target ALS Global Natural History Study: https://bit.ly/3Y2ROSS #ALSResearch #ALS #GlobalNaturalHistoryStudy #Diversity

New Funding Opportunity! Target ALS is excited to announce a new grant opportunity for consortia developing novel modalities as therapeutics for ALS. This funding call for collaborative groups aims to facilitate the discovery and development of molecules targeting RNA/DNA to drive innovation in ALS treatment. What We're Funding: - Development of new antisense oligonucleotide chemistries, CRISPR, Zinc Finger, siRNA, and more. - Enhanced brain delivery platforms like lipid nanoparticles, AAV, and others. - Molecules with demonstrated cell-based activity needing additional validation - Modalities applied to an ALS-associated genetic target Key Dates: LOI due: November 18, 2024 Full proposals due: February 21, 2025 Funding begins: May 2025 This grant is an exciting opportunity to collaborate with academic and industry partners to accelerate breakthroughs in ALS research. Learn more and apply: https://bit.ly/4eQHsMl #ALSResearch #GrantOpportunity #GeneTherapies #InnovativeScience #TargetALS #EveryoneLives

Meet Kylee and Rodney Dial, from Palo Alto, California, who are taking on the TCS New York City Marathon together in support of #TeamTargetALS Their journey with ALS began when they became friends with Jeni and Stephen, another couple expecting a baby at the same time. Stephen, diagnosed with ALS at just 27, was already several years into his battle when they met. Despite the challenges, the two families have shared countless memories together, from watching their little ones grow to cheering on their favorite sports teams. Rodney and Kylee have long dreamed of running the iconic New York City Marathon, and in 2024, their dream will come true as they move to NYC for a year. Their excitement for the race is matched only by their passion for raising money and awareness for ALS, a disease that has deeply touched their lives through their friendship with Stephen. “We felt so lucky to be introduced to Target ALS, an innovative medical research foundation committed to the search for effective treatments for ALS. The opportunity to raise money and awareness for ALS, a disease that plagues one of our closest friends, as we train for and run this race is so meaningful. We cannot wait to cross the finish line in November (with a slice of pizza to celebrate, of course!),” say Rodney and Kylee. Support Rodney and Kylee as they run to create a world where Everyone Lives. Every donation brings us closer to finding effective treatments for ALS: https://bit.ly/4eKxVqo #ALSResearch #NYCMarathon #EveryoneLives #DonateNow

Milestone Achieved: 8th Clinical Trial Emerges from Target ALS funded consortia QurAlis has completed dosing the first participant cohort in a Phase 1 clinical trial for QRL-101, a first-in-class precision therapy targeting hyperexcitability in ALS. This breakthrough treatment has the potential to benefit both familial and sporadic ALS patients by addressing motor neuron degeneration caused by hyperexcitability—affecting nearly 50% of all cases. Target ALS is proud to have supported the foundational research that led to this clinical trial, as well as providing additional grants, marking a significant step toward effective treatments for ALS. Read the full press release here: https://prn.to/47KVTiX #ALSResearch #ClinicalTrial #EveryoneLives #MilestoneAchieved

Exciting new research, utilizing the Target ALS Human Postmortem Tissue Core, has revealed that prion-like SOD1 protein aggregates are present in ALS patients without a SOD1 gene mutation. This breakthrough, led by Dr. Moses Leavens from the McLaughlin Research Institute for Biomedical Sciences, suggests that diagnostic biomarkers for familial ALS may also benefit sporadic ALS cases, which account for 90% of ALS diagnoses. With support from Target ALS, ongoing studies aim to validate these findings and advance diagnostic tools. Read the full blog and published work here: https://bit.ly/4dv9a02 Learn more about how our resources are accelerating ALS research at low to no cost to scientists worldwide: https://bit.ly/4dvk1HB #EveryoneLives #ALS #ALSResearch

Meet Alexandria Winkler one of our inspiring runners for the TCS New York City Marathon! From a young age, Alex grew up cheering on her dad during Ironmans, half marathons, and marathons, shaping her into the determined and passionate person she is today. Now, it's her turn to hit the pavement, running her first marathon in honor of her beloved grandfather, Gordon Petrie, who bravely fought ALS for seven years. “I am so honored that I have the opportunity to help raise money for ALS research. ALS took our grandfather away from us, but we still keep him close,” says Alex. Join Alex on her journey and help support her mission to create a world where Everyone Lives. Every donation brings us one step closer to finding effective treatments for ALS: https://bit.ly/3Xhpuvr #TeamTargetALS #ALSResearch #NYCMarathon #EveryoneLives #DonateNow

Target ALS is partnering with Modality.AI to launch a groundbreaking direct-to-participant study, utilizing Modality's innovative digital health platform to monitor disease progression in ALS patients. The study, part of our Global Natural History Study, will recruit 150 participants, with 2 already enrolled, across five international sites, including Spanish-speaking countries. Modality’s AI-driven platform uses a virtual guide to capture participants' speech and facial biomarkers, activities of daily living such as brushing their hair and washing their face, and various cognitive tasks such as word recall and sequential movements, providing a self-directed assessment from the comfort of participants' homes. This approach promises to enhance patient diversity, retention, and the sensitivity of tracking ALS progression. “Modality.AI's technology is at the forefront of digital health technologies being evaluated for the utility of predicting and tracking disease progression in ALS patients,” says Amy Easton, our Senior Director of Scientific Programs. "These technologies are being developed to allow clinicians, patients, and caregivers to measure disease symptoms more sensitively and reliably than standard clinical assessments, and in the future, may even replace clinical outcome assessments used to evaluate novel treatments for ALS." Learn more about this innovative collaboration here: https://bit.ly/4eBNIYm Research from this pioneering study will be made available on the Target ALS Data Engine: https://bit.ly/3TJk5ME #ALSResearch #DigitalHealth #ModalityAI #TargetALS #EveryoneLives