Target ALS

09/10/2026

Clinical trials can feel confusing, even overwhelming, especially when you or someone you love is facing an ALS diagnosis. Our next webinar is here to help.

Join us on September 21 for "Understanding Clinical Trials: A Guide for the ALS Community," featuring Dr. Toby Ferguson and Dr. Thos Cochrane, moderated by Stephanie Ishoo.

We'll cover what makes a strong drug target, how a discovery program becomes a clinical trial, what happens in each trial phase, how to understand primary and secondary endpoints, and lessons from the tofersen trial for SOD1 ALS. We'll also discuss the questions you should ask before deciding to participate in a trial.

Bring your questions. There will be time for audience Q&A.

Register now: https://ow.ly/GqKq50ZMjWU

09/10/2026

ALS research has a diversity problem. Historically, only 7% of participants in ALS clinical research studies have been non-Caucasian, which means we don’t have a full picture of the disease.

We’re changing that with the ALS Global Research Initiative (AGRI), a worldwide effort to speed up discovery by removing barriers. No embargoes. No waiting periods. Participant samples and data are shared with scientists across the globe almost immediately.

Through our Community-Based Outreach Study (CBOS) and our Global Natural History Study (GNHS), AGRI is tracking ALS progression, identifying biomarkers, and building a more representative collection of samples and data, with a third of participants now coming from diverse backgrounds.

Faster science. Broader access. Better treatments for everyone.

Learn more about AGRI and how to get involved: https://ow.ly/I3gZ50ZLHPR

09/04/2026

Meet Katie Daigle, a Doctor of Physical Therapy student running the 2026 TCS New York City Marathon with Team Target ALS.

Katie's grandfather, Harold Haydel, a former MLB pitcher for the Minnesota Twins, battled ALS from 2012 to 2018. Katie remembers the day his arms grew too weak to throw a baseball with her cousins, and using an alphabet board so he could blink out what he wanted to say. Watching someone who built a career on physical strength lose his ability to move and communicate showed Katie at a young age just how devastating this disease can be.

Now studying to become a physical therapist, Katie has seen firsthand the difference compassionate care can make for people living with ALS, and it has only deepened her drive to advocate for this community, both in and out of the clinic.

Every mile Katie trains and races is dedicated to those fighting ALS, those who have lost their battle, and the families who stand beside them.

Support Katie's run and help us build a world where Everyone Lives: https://ow.ly/KZkL50ZItG5

09/02/2026

Next month, we're heading to the Network of Excellence for ALS - NEALS Annual Meeting, one of the largest gatherings of ALS researchers, clinicians, and industry partners working to speed up the path from discovery to treatment.

This year, we're proud to bring 8 accepted abstract posters, representing work across our ALS Global Research Initiative (AGRI), Global Natural History Study (GNHS), and our Postmortem Tissue Core (PMTC).

🧠 3 posters
🧬 1 poster
🤝 2 coauthored posters with Modality AI and AMP® ALS
🔬 2 posters

As Laura Dugom shared, with this growth comes more data, more science, and more opportunities to learn, share, and accelerate progress for people living with ALS. These growing resources are helping researchers uncover new biological insights, identify biomarkers and potential therapeutic targets, and ultimately design better, more informative clinical trials.

Laura and Marina Selenica from our team will be attending in person. If you're at NEALS this year, come find them and say hello!

08/31/2026

Join us for the final installment of North Star ALS' ILLUMINATE: Precision Medicine webinar series on Thursday, September 3.

This session, "Looking ahead," brings together Jeffrey D. Rothstein, MD, PhD (Robert Packard Center for ALS Research, Answer ALS), Thos Cochrane, MD, MBA (former Global Medical Lead for ALS at Biogen), our own Laura Dugom, MPH (Associate Director, Clinical Research, Target ALS), and Jean Swidler (Genetic ALS & FTD: End the Legacy).

The panel will dig into how precision medicine is reshaping ALS drug development, what longitudinal data reveals about disease progression, when a biomarker becomes truly actionable, and what it will take to move from earlier intervention toward prevention.

Register here: https://ow.ly/hVws50ZHteQ

Illuminate: Precision Medicine includes four webinars moderated by Dr. Nadia Sethi. Join us as we explore how ALS research is advancing toward targeted therapies, featuring insights from leading researchers, clinicians, and individuals with lived experience. ALS is often described as heterogeneous,....

08/28/2026

Meet Amanda, a two-time marathoner taking on the New York City Marathon this year in support of ALS research and advocacy.

For Amanda, this cause is personal. Her friend Alexandra, president of Her ALS Story, is living with ALS, and Amanda has seen firsthand the physical and emotional toll the disease takes and the reality of facing an illness medicine still can't stop.

"Running 26.2 miles is hard," Amanda says. "But it is nothing compared to what Alexandra navigates every single day."

Every mile in New York will be dedicated to Alexandra, and to everyone fighting ALS without enough research funding, treatment options, or awareness behind them. Amanda is committed to training with purpose, fundraising with intention, and using her platform to share why she's running with everyone who asks.

Help Amanda reach the finish line and support her fundraising goal. Every donation moves us closer to a world where Everyone with ALS Lives: https://ow.ly/Thhl50ZFVEV

08/26/2026

Dr. Johnathan Cooper-Knock and his team set out to identify a genetic modifier of how long a person with ALS lives with the disease, because ALS progression varies so widely.

He's seen it firsthand in his clinical practice: a brother and sister, both with , whose disease duration differed by 20 years. His takeaway is a message of hope: if we can understand what turns 6 months into 20 years, that's the blueprint for a drug that works.

To get there, his team used an AI model to understand the genome specifically in motor neurons, the cell type affected in ALS, uncovering signals standard techniques couldn't reach.

But is only as powerful as what feeds it. It can speed up analysis, but it cannot replace biosamples, patient data, and tissue. None of that can be simulated.

That's where Target ALS comes in. Our and build the foundation researchers like Dr. Cooper-Knock need to put AI to work for the ALS community. Read more about how we're building that foundation: https://ow.ly/EU2R50ZFVvp

08/21/2026

Meet Joe Mathew, who's lacing up for his second marathon, but his first time running for a cause.

Joe's connection to ALS is personal. His uncle is living with the disease, and Joe is running to raise awareness.

This isn't Joe's first time supporting Target ALS either. Six years ago, he raised nearly $1,000 for us through the Ice Bucket Challenge. Now he's back with an even bigger goal: to raise six times that amount, with his family cheering him on every step of the way.

Help Joe reach the finish line and his fundraising goal. Every donation brings us closer to effective treatments and a world where Everyone Lives:
https://ow.ly/WWpR50ZCAps

08/19/2026

Evangelos Kiskinis describes this moment as the most exciting time in ALS research, and he'd know. When he started in this field, scientists understood just one gene linked to ALS. Today, that number has grown past 30.

But new genetic findings are only part of the story. In this video, Dr. Kiskinis reflects on everything that's changed around him: the resources now available for ALS research, the diverse disciplines and investigators drawn into the field, and the clinical trials that have gone from limited to many. Most importantly, these trials are rational, built on mechanisms the field has spent years working to understand.

Explore the progress: https://ow.ly/XfKR50ZBwfA

08/19/2026

Tomorrow, Target ALS VP of Scientific Programs, Amy Easton, PhD, will join a panel on Research & Industry: Building Precision Therapies, part of North Star ALS's Illuminate: Precision Medicine series.

📅 August 20, 2026
🕐 1:00 PM PST / 4:00 PM EST

Amy will be joining:
Neil Shneider, MD, PhD
Eric Green, MD, PhD
Olga Uspenskaya, MD, PhD

The panel will dig into the ALS treatment pipeline, the role of omics in drug development, and how precision therapeutics are reshaping what's possible for the 97% of ALS cases without a known single genetic cause.

Register here: https://ow.ly/KNsQ50ZBvZp