Target ALS

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Founded in 2013 by Dan Doctoroff, Target ALS is a medical research foundation whose mission is to accelerate discovery to find effective treatments and ultimately build a world where Everyone with ALS lives.

04/15/2026

Martha Peña Preciado is a neurologist at Instituto Roosevelt in Bogotá, Colombia, and has worked with people with amyotrophic lateral sclerosis (ALS) and their families for 20 years. Dr. Peña Preciado is a Principal Investigator for Target ALS’s Global Natural History Study because she believes that this project will advance our understanding of how ALS develops and how we can intervene. Latin American populations are highly diverse and differ from North American and European populations. We must study ALS across all populations to identify treatments that are effective for everyone.

We are grateful to be working with Dr. Peña Preciado and her team on this project.

More information about the Global Natural History Study here: https://targetals.org/global-natural-history-study/

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Martha Peña Preciado es neuróloga en el Instituto Roosevelt en Bogotá, Colombia, y ha trabajado con personas con esclerosis lateral amiotrófica (ELA) y sus familias durante 20 años. La Dra. Peña Preciado es una investigadora principal del Estudio Global de Historia Natural de Target ALS porque cree que este proyecto va a avanzar en cómo se genera la ELA y cómo podemos intervenir. Las poblaciones latinoamericanas son muy diversas y tienen diferencia con las poblaciones norteamericanas y europeas. Debemos estudiar la ELA en todas las poblaciones para identificar tratamientos que sean eficaces para todos.

Estamos agradecidos de trabajar con la Dra. Peña Preciado y su equipo en este proyecto.

Más información sobre el Estudio Global de Historia Natural aquí: https://targetals.org/global-natural-history-study/

Our most-attended webinar yet, and the playback is now live.Over 360 registrants from 18 countries tuned in to hear Targ...
04/14/2026

Our most-attended webinar yet, and the playback is now live.

Over 360 registrants from 18 countries tuned in to hear Target ALS VP of Research Amy Easton, PhD, DNAstack CEO Marc Fiume, PhD, and Michael J. Fox Foundation VP of Research Data Resources Leslie Kirsch, EdD, discuss how big data is transforming neurodegenerative disease research.

The conversation covered how large-scale data sharing is accelerating the path to treatments for ALS, Parkinson’s Disease, and more, and why it matters now.

🎥 Watch the full playback: https://ow.ly/yT4Z50YJ4aI

36 publications. One quarter. One shared mission to change what it means to receive an ALS diagnosis. Researchers across...
04/14/2026

36 publications. One quarter. One shared mission to change what it means to receive an ALS diagnosis. Researchers across our innovation ecosystem, the global network of scientists we fund and enable, are moving fast.

View the full list: https://ow.ly/fG0150YJ3Ej

Target ALS is proud to announce a newly funded 3-year precompetitive consortium: "C9orf72 disease drivers: From genetic ...
04/10/2026

Target ALS is proud to announce a newly funded 3-year precompetitive consortium: "C9orf72 disease drivers: From genetic modifiers to toxic mechanisms."

Despite years of research and multiple clinical trials, the field is still working to understand why disease presentation in C9orf72 ALS, the most common genetic cause, varies so dramatically between individuals, families, and even across tissues in the same person.

This powerhouse consortium brings together world-class expertise in C9orf72 genetics and molecular biology. All data will be shared through the Target ALS Data Engine, open to researchers in academia and industry, and will contribute to our growing database from C9orf72 carriers.

Read more: https://ow.ly/2guu50YEGGi

We're now accepting applications for grants to collaborative consortia studying ALS biomarkers of disease stratification...
04/07/2026

We're now accepting applications for grants to collaborative consortia studying ALS biomarkers of disease stratification and progression.

As new therapeutics move the needle for those living with ALS, the need to track disease progression and monitor treatment response has never been greater.

Target ALS is seeking multidisciplinary groups of 3–5 labs, which include at least one industry partner, to develop novel fluid or tissue-based biomarkers, including approaches using AI and machine learning.

LOI deadline: May 19, 2026. Apply today: https://ow.ly/WYix50YEGxE

Without investment in early-stage science, there would be no clinical trials. Target ALS funded a preclinical study led ...
04/06/2026

Without investment in early-stage science, there would be no clinical trials.

Target ALS funded a preclinical study led by QurAlis that tested Stathmin-2 (STMN2), a protein important for muscle innervation that is downregulated in ALS, as a drug target.

Recently, the biotech company has reported positive interim data from their ANQUR study, the first-ever clinical trial aimed at STMN2 restoration in ALS. Their lead candidate, QRL-201, elevated STMN2 levels, showed favorable safety, and reduced pNFH, a biomarker of motor neuron degeneration, compared to placebo.

This is what fueling the pipeline looks like. And we’re proud to have been there from the beginning. Dive deeper: https://ow.ly/QbYq50YEG3t

Two years ago, we opened the doors to the Target ALS Data Engine. Today, we're celebrating what we’ve built and how it’s...
04/02/2026

Two years ago, we opened the doors to the Target ALS Data Engine. Today, we're celebrating what we’ve built and how it’s fueling discoveries across the global research community.

We didn’t want to simply create another platform. We aimed to change the culture of ALS research from siloed and slow to open, accessible, and fast. Every dataset shared, every researcher who logs in, and every analysis run is a direct step toward a better future for people living with ALS, many of whom make this work possible by generously donating biosamples.

The best part? We're just getting started. Swipe to see what two years of data sharing and open-access ALS research looks like.

Learn more about the Target ALS Data Engine: https://targetals.org/research/genomic-datasets/

ALS did not enter this family once and leave. It came back again and again, taking a father, a brother, then two sisters...
03/31/2026

ALS did not enter this family once and leave. It came back again and again, taking a father, a brother, then two sisters.

In this deeply personal story, Lynn Koll shares the journey of her three sisters, Anne, Jan, and Lora. From early loss to genetic discovery and the weight of knowing what may come, their lives have been shaped by this fatal disease. And still, they all chose to act. To advocate, to participate in research, and to push for a different future.

We are deeply grateful to Lynn for sharing her family’s story and shining a light on the reality of . Stories like these keep all of us moving forward with impatient optimism for the future so that one day no family has to endure loss like this. Together, we will build a world where .

Read the full story: https://ow.ly/mMA850YzspM

Progress in ALS doesn’t happen in isolation. It happens when collaboration replaces silos, and shared infrastructure acc...
03/30/2026

Progress in ALS doesn’t happen in isolation. It happens when collaboration replaces silos, and shared infrastructure accelerates discovery.

That philosophy is at the heart of the Accelerating Medicines Partnership® ALS (AMP® ALS), a large-scale public-private effort designed to accelerate the development of biomarkers and effective treatments for ALS.

Target ALS has been involved from the outset, with founder Dan Doctoroff and CEO Manish Raisinghani working together with Walter Koroshetz, former Director of the National Institute of Neurological Disorders and Stroke (NINDS), and Alessio Travaglia, Director, Translational Science, Neuroscience and Rare Diseases, FNIH, to help shape how resources could be used most effectively. Dan was a unanimous choice to serve as Co-Chair of the AMP ALS Steering Committee, alongside leaders from across the partnership.

Beyond the design phase, Target ALS also drives ex*****on of the program today. Target ALS VP of Research Amy Easton works closely with Amelie Gubitz and Amy Tsou (both Program Directors at NINDS), David Shulman (person living with ALS), and Shubhangi Lal (Senior Project Manager, Neuroscience, FNIH) to co-lead the ALL ALS natural history study Working Group, which provides critical guidance for ALL ALS.

Target ALS is proud to play a central leadership role in advancing research alongside partners from government, industry, academia, nonprofits, and people with lived experience of ALS.
This is what it looks like when the entire ecosystem moves forward together.

Read more about our role in AMP® ALS: https://targetals.org/news/accelerating-progress-together-target-alss-role-in-amp-als/

03/30/2026

We’re proud to recognize the women leading some of our Global Natural History Study ( ) sites that are advancing around the world.

As part of the ALS Global Research Initiative ( ), these sites and women play a critical role in building the most comprehensive, representative studies ever assembled. They are enrolling participants, collecting longitudinal data and biosamples, and ensuring research reflects the full diversity of the ALS community, across geographies, backgrounds, and lived experiences.

Their leadership strengthens the foundation of ALS research and brings us closer to discoveries that can translate into meaningful progress for people living with this disease.

We’re grateful for the women leading this work globally and helping reshape what’s possible for ALS research.

Read more features and submit a story of your own: https://ow.ly/V2Po50Yzsoa

Erin Cox was about to start the next chapter of her life when her dad was diagnosed with ALS. Instead, she put everythin...
03/28/2026

Erin Cox was about to start the next chapter of her life when her dad was diagnosed with ALS. Instead, she put everything on hold and stepped into a role she never expected, becoming his primary caregiver.

Caregiving for someone with ALS is not just the big moments. It is the quiet, constant, everyday details that never stop. It is learning routines, anticipating needs, and carrying the weight of a new reality while trying to preserve a sense of normalcy for someone you love.

We are incredibly grateful to Erin’s sister, Emily, for sharing Erin’s story and honoring the caregivers who show up every single day in ways that often go unseen. Stories like this remind us what ALS truly looks like and why the work to change it matters.

Read the full story: https://ow.ly/nWl950Yzsec

Tiffany Dillon is 31 years old, eight months pregnant, and living with . She's a former Division I athlete who built he...
03/27/2026

Tiffany Dillon is 31 years old, eight months pregnant, and living with . She's a former Division I athlete who built her life around movement and has spent the past year redefining what that means.

Her diagnosis came after months of uncertainty: a stiff foot, unexplained falls, and nearly eight months of tests before finally getting answers. Rather than stepping back, Tiffany stepped forward by raising funds for Target ALS and investing in the research that could change what this disease looks like for everyone who comes after her.

This , her story is a reminder that strength isn't always loud. Sometimes it's showing up, adjusting, and continuing forward, even when things don't look the way you thought they would.

🔗 Read her full story and support her fundraiser: https://ow.ly/BVoY50YzUXK

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