Target ALS

Target ALS Founded in 2013 by Dan Doctoroff, Target ALS is a medical research foundation whose mission is to acc

Today, on International Day of Women and Girls in Science, we celebrate the women driving discovery forward: asking bett...
02/11/2026

Today, on International Day of Women and Girls in Science, we celebrate the women driving discovery forward: asking better questions, building better models, and making strides toward effective treatments for ALS.

When science reflects the full breadth of talent and perspective, progress accelerates. For ALS, that progress can’t come soon enough.

Expanding where we conduct research opens up a whole new world of information about ALS. We’re adding four new sites in ...
02/10/2026

Expanding where we conduct research opens up a whole new world of information about ALS.

We’re adding four new sites in Malaysia, Florida, and India to the Global Natural History Study, a cornerstone of our ALS Global Research Initiative (AGRI). By expanding our efforts into new communities around the world, we’re strengthening the global foundation needed to understand ALS across diverse populations and environments. These sites will help ensure the data driving discovery is more representative, more powerful, and more actionable.

Stay tuned for updates on when these new sites begin accepting participants.

Find your nearest site and learn more about AGRI at www.targetals.org/agri

Better models mean better answers and faster progress toward treatments.Target ALS has partnered with Regeneron to enabl...
02/10/2026

Better models mean better answers and faster progress toward treatments.

Target ALS has partnered with Regeneron to enable access to a novel mouse model of and driven by repeat expansions, the most common genetic cause of both diseases. By removing IP and data-sharing barriers, this partnership gives researchers across academia and industry the freedom to rigorously test new therapeutic ideas using a disease-relevant model.

This is how we accelerate discovery: by sharing the right tools, at the right time, with the entire ALS community.

Read more about the partnership: https://ow.ly/uL1950Yc38x

Turning discoveries into real treatments is often the hardest challenge in .After nearly seven years of rigorous scienc...
02/06/2026

Turning discoveries into real treatments is often the hardest challenge in .

After nearly seven years of rigorous science and validation, NRG Therapeutics has dosed the first participants in a Phase 1 clinical trial of NRG5051, a novel therapy designed to stabilize mitochondria, the cell’s energy producers, which are dysfunctional in ALS.

Target ALS supported this work at a critical point, helping answer a make-or-break question: could stabilizing mitochondrial function meaningfully protect neurons in ALS-relevant models? By enabling access to the right mouse model and removing barriers that slow translation, this research moved from promising biology to a real clinical trial candidate.

This milestone shows why early, deliberate investment matters. It is how ideas move beyond the lab and closer to people living with .

Read the full story: https://ow.ly/HczT50YaoCm

02/04/2026

What is ALS?

ALS (amyotrophic lateral sclerosis) is a progressive neurodegenerative disease that affects motor neurons, the nerve cells responsible for muscle movement. Over time, these motor neurons stop working, leading to muscle weakness, loss of mobility, and eventually the inability to speak, eat, or breathe independently.

Today, approved treatments have only a limited benefit or apply to a subset of people with ALS. But through bold, collaborative research, Target ALS is working to better understand the disease, identify biomarkers, and develop effective treatments so everyone with ALS lives.

Learn more at https://ow.ly/Poag50Y6FRC

Breaking down data barriers accelerates discovery.The Target ALS Data Engine was built to give researchers immediate acc...
02/03/2026

Breaking down data barriers accelerates discovery.

The Target ALS Data Engine was built to give researchers immediate access to open, harmonized, human-derived ALS datasets, no egregious paywalls, no IP restrictions, no delays.

Today, more than 84 industry groups, including GSK’s neurodegeneration research team, are using the Data Engine to advance target identification, biomarker discovery, and translational research.

As Dr. Sanjay Kumar of GSK shared, the platform is already informing work across genomics, transcriptomics, and emerging long-read sequencing approaches.

This is what science looks like when data are shared without friction.

🔗 Read more: https://ow.ly/I1UP50Y6FQE

After being diagnosed with ALS at just 31, Tiffany Dillon was faced with a life-altering reality. Tiffany launched a fun...
02/02/2026

After being diagnosed with ALS at just 31, Tiffany Dillon was faced with a life-altering reality.

Tiffany launched a fundraiser to support ALS research, driven by a deep belief in progress, in the scientists working tirelessly for answers, and in a future where ALS can be effectively treated and ultimately prevented. Her story is a powerful reminder that even in the face of uncertainty, there is room for hope, agency, and impact.

DIY fundraising is about meeting the moment in your own way: honoring a loved one, celebrating a milestone, or simply standing with the ALS community.

Start your own fundraiser, your way, and help move ALS research forward: https://ow.ly/jP6350Y5aog

You can also support Tiffany’s fundraiser here: https://ow.ly/CysV50Y5aoi

Why do some people with ALS-linked genetic mutations develop the disease while others remain symptom-free?Target ALS is ...
01/29/2026

Why do some people with ALS-linked genetic mutations develop the disease while others remain symptom-free?

Target ALS is supporting a new global research effort led by Dr. Juliana Acosta-Uribe to answer that question by studying large, multigenerational families carrying a TARDBP (TDP-43) mutation associated with ALS in Medellín, Colombia.

By working directly with families and comparing affected and unaffected individuals, this study aims to uncover protective factors that may delay or even prevent .

This community, field-driven approach reflects Target ALS’s commitment to advancing science through collaboration and compassion, looking toward a future with targeted and preventative therapies for ALS.

Read more about the TARDBP study: https://ow.ly/sYza50Y5aab



Grupo de Neurociencias de Antioquia

For years, inflammation has been considered a potential contributor to . But what if, for some people, the problem isn’...
01/28/2026

For years, inflammation has been considered a potential contributor to . But what if, for some people, the problem isn’t too much immune activity, but too little?

New research from Target ALS–funded investigator Dr. Pegah Masrori, published in Nature Neuroscience, shows that in C9orf72-linked ALS, the most common genetic form of the disease, the brain’s immune cells fail to fully respond when neurons are under stress.

These findings challenge long-held assumptions and help explain why ALS is not a one-size-fits-all disease. By investing in this kind of basic biology-driven research, Target ALS is helping uncover new pathways toward effective treatments.

Read more: https://ow.ly/6kZI50Y4BRn

By directly conducting our own clinical studies, we maintain consistency, improve data quality, and adapt quickly as new...
01/27/2026

By directly conducting our own clinical studies, we maintain consistency, improve data quality, and adapt quickly as new questions emerge. This model helps ensure research is both rigorous and responsive to the needs of the ALS community.

Bringing opportunities to participate in research directly into communities and uniting efforts across borders, we are building a clearer, more complete picture of ALS: one that supports earlier diagnosis, better tracking of disease progression, and more informed clinical decisions.

Read more about the research we conduct: https://ow.ly/9k0o50Y4BJb

Through the ALS Global Research Initiative (AGRI), Target ALS is expanding who can participate in research and what that...
01/26/2026

Through the ALS Global Research Initiative (AGRI), Target ALS is expanding who can participate in research and what that research can deliver.

In 2025, AGRI data reflected meaningful gains in representation, with more than one-third of participants identifying as non-white and of non-European descent, and biofluids are already fueling new biomarker discoveries to support earlier diagnosis and stronger clinical trials.

This is how we build the foundation for effective treatments for everyone living with .

Read more in our 2025 Year in Review: https://ow.ly/R9Ig50Y3VR0

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