The numbers behind ALS tell a powerful story, illuminating the urgent need for progress in combating this life-altering disease. With each new study, researchers uncover vital information about risk factors, genetic links, and potential pathways for treatment. By understanding the data, we can amplify our efforts to raise awareness, provide meaningful support to the ALS community, and drive the innovations needed to one day realize a world where everybody lives.
Uncovering ALS’s Hidden Impact
Amyotrophic Lateral Sclerosis (ALS) may not dominate news cycles, but its devastating impact reverberates through countless lives. Numbers only tell part of the story. Behind each ALS disease statistic lies a person, a family, and a community significantly impacted.
The economic toll is staggering, with care costs often exceeding $250,000 annually. The emotional cost? Immeasurable.
ALS Disease Statistics: An Essential Overview
ALS, or amyotrophic lateral sclerosis, affects nerve cells responsible for muscle action. As a result, muscles begin to waste away, causing weakness.
Each year, ALS is diagnosed in about 1.7 to 2.2 out of every 100,000 people. These numbers make it a rare illness in the broader scope of health conditions. Although rare, its impact on those affected and their families is profound.
Difficulty of Diagnosing ALS
ALS symptoms can mimic those of other neurological diseases, such as dementia, multiple sclerosis, or peripheral neuropathy. Early symptoms, like muscle weakness and cramps, are often subtle and can be easily attributed to aging or other conditions.
There is also no single test for ALS, and diagnosis typically relies on a combination of clinical evaluations, history, and the exclusion of other conditions. This often involves a series of tests to assess muscle and nerve function.
ALS symptoms can also progress at different rates, making it difficult to determine if a person is experiencing a degenerative disease or a temporary condition. This variability can lead to delays in diagnosis.
These factors contribute to the complexity and difficulty of diagnosing ALS, often resulting in a lengthy diagnostic process.
Types of ALS
ALS is divided primarily into two types: Sporadic and Familial.
Sporadic ALS
Sporadic ALS is the most common, representing 90-95% of all cases. It arises seemingly randomly, with no known family history. This means many cases develop without an apparent cause, making research into its origins challenging. Gene mutations can cause sporadic ALS by triggering abnormal protein folding or cellular dysfunction, even in the absence of a family history, due to random genetic changes or environmental factors that affect the nervous system.
Familial ALS
Familial ALS accounts for about 5-10% of all cases. Unlike sporadic ALS, familial ALS is genetic and often passed through families. Genetic mutations, such as those found in the SOD1 gene, are contributing factors.
Factors influencing ALS disease progression rates
ALS rates depend on several factors, including genetics, environment, and lifestyle. A strong genetic predisposition can be present, as seen in familial ALS cases. Gene mutations like those in the C9orf72 gene significantly influence these cases. In contrast, sporadic ALS might find its roots more in environmental aspects.
There’s been considerable interest in the link between ALS and military service. Studies suggest veterans might face a higher ALS risk. You can learn more about veterans and ALS here.
ALS Demographic Trends
Age-related trends in ALS
Most ALS diagnoses occur between the ages of 40 and 70. This age range reflects the majority of cases, making it a focal point for understanding disease onset and progression. Interestingly, ALS is extraordinarily rare in individuals younger than 20. As age progresses, risks associated with cellular degeneration become more pronounced, complicating the body’s ability to repair nerve cells.
Neuroscience research highlights this connection between aging and ALS. These studies delve into cellular mechanisms like oxidative stress and malfunctioning protein aggregation that worsen with age.
The age factor triggers questions on whether the late-life onset of ALS could mask underlying vulnerabilities accumulating over a lifetime. While genetics play a key role, lifestyle factors such as dietary habits and physical activity levels may also influence age of onset.

Gender Differences in ALS Incidence
Although ALS affects both genders, men slightly outnumber women. The ratio often seen is 1.5 males for every female diagnosed.
Social behavior potentially affects gender-based differential exposure to risk factors as well. Occupational hazards and lifestyle choices may disproportionately impact men, elevating incidence rates. While this aspect remains under investigation, industrial exposure studies hint at occupational risks playing a role.
Ethnic and Geographical Variations
ALS incidence varies significantly among different ethnic groups and regions. Caucasians show higher rates of ALS compared to other ethnic groups and the C9orf72 gene mutation is more frequent in European populations. However, a lack of research in diverse populations and underrepresented communities impedes progress in understanding the global incidence and cause of ALS and creates bias in research that is currently available. The Target ALS Natural History Study’s comprehensive biosample and dataset collection for ALS biomarker discovery and research efforts aims to address these gaps in data. The consortium is expanding to include international sites to bolster sample analyses from diverse genetic and ethnic backgrounds. Data will provide insight into the cause of the disease for everyone. Learn more here.
Advances in ALS Studies and Genetic Research
Genetic research into ALS has made significant strides recently. New genes associated with ALS have surfaced, offering promising avenues for diagnostics and treatment. For example, a recent study published by members of a Target ALS research consortium explores the genetic underpinnings of the disease using machine learning analysis of large datasets. Such discoveries hold potential for targeted therapeutic approaches and advanced diagnostics in affected populations.
C9orf72, SOD1, FUS, and TDP-43 are four key genes associated with ALS, each playing a role in the disease’s pathogenesis. The discovery of SOD1 mutations in the 1990s marked a significant breakthrough, followed by the identification of FUS mutations in 2009 and C9orf72 mutations in 2011. Mutations in these genes which increase the risk of developing ALS impact numerous cellular processes in neurons and other cells in the body. The accumulation of TDP-43 protein in ALS patients is a hallmark of both sporadic and familial cases of the disease. In rare cases, mutations in the TDP-43 gene can run in families and cause ALS. Together, these genes and their discovery highlight the importance of understanding the genetic basis of the disease, as these breakthroughs can pave the way for novel disease-modifying therapeutics. Understanding the genetic basis of the disease can also drive additional understanding of ALS biology and provide new avenues for biomarkers.
Further investigations have also highlighted the ARPP21 gene, particularly in a localized region in Spain. NEK1 mutations have been linked to disrupted motor neuron function, another step forward in understanding ALS at a molecular level, in research supported by Target ALS.
Genetic research plays a pivotal role in uncovering disease mechanisms, enabling targeted approaches to therapy and diagnostics, and fostering a global understanding of ALS. Initiatives such as the Global Neurogenetics Health Study (GNHS) and Outreach studies are instrumental in examining the genetic nature of ALS across diverse populations. By investigating how genetic variations manifest around the world, these studies lay the groundwork for implementing therapies that aim to benefit everyone, regardless of their genetic background. Through this inclusive approach, we advance the vision of ensuring that Everyone Lives.
“If we find a genetic change, we know it’s a driver of the disease. It’s not a consequence of disease. And that’s really exciting because if we can correct that upstream driver, then potentially we’ve got a therapy.” – Dr. Johnathan Cooper-Knock, Clinician Scientist at The University of Sheffield.
Target ALS has been making strides in funding research around future diagnosis and treatment for all forms of ALS. This includes:
- Research focused on understanding protein aggregation biology and developing seed amplification assays (SAAs) as diagnostic tools for ALS.
- Phosphorylated TDP-43 (pTDP-43) aggregates are a hallmark of 97% of ALS cases, yet pTDP-43 antibodies have historically been unreliable or difficult and expensive to license. As part of our mission to break down barriers to ALS research, Target ALS has worked with Len Petrucelli and DSHB to provide a validated pTDP-43 antibody to the global ALS research community.
- Two Target ALS-funded consortia have now published work that could usher in the first ALS diagnostic biomarker. In partnership with Biogen, Phil Wong from Johns Hopkins University identified an extra piece of protein (“cryptic peptide”) in the HDGFL2 protein. This cryptic peptide appears without functional TDP-43, an RNA-binding protein whose dysfunction is implicated in 97% of ALS cases. In a recent Nature Medicine publication, the consortium reported detection of this HDGFL2 cryptic peptide in postmortem tissue from individuals with ALS and FTLD, where it specifically appeared in neurons also lacking functional TDP-43. Further exploration of the HDGFL2 cryptic peptide in cerebrospinal fluid of individuals with familial and sporadic ALS revealed this cryptic peptide appears prior to ALS symptoms in people with ALS and FTD-related C9orf72 repeat expansion and is also present in individuals with sporadic ALS.
These breakthroughs present the possibility of significant progress in creating new therapies that target specific genetic pathways involved in ALS. Such genetic insights highlight not only the complexity of ALS but also the nuances involved in developing precision medicine strategies. Target ALS is expanding our portfolio of supported genetic therapies in 2025 through our Consortia researching New Modalities targeting DNA/RNA Research Funding Announcement. Stay tuned for more details.
Emerging Treatments and Therapies
Therapeutic development for ALS is gaining momentum. Researchers are exploring drugs that could slow down disease progression, including the OurAlis hyperexcitability trial. This treatment approach draws from existing medications used in autoimmune diseases, aiming for quick repurposing.
Another breakthrough is Tofersen, an antisense oligonucleotide therapy specifically designed to target the SOD1 gene mutation, which is associated with a subset of ALS cases. Recent trials have shown that Tofersen can significantly reduce levels of harmful SOD1 protein, offering hope for individuals with this genetic form of ALS.
Therapeutic development for ALS is gaining momentum. Researchers are exploring drugs that could slow down disease progression, including the OurAlis hyperexcitability trial. This treatment approach draws from existing medications used in autoimmune diseases, aiming for quick repurposing.
Machine learning has identified new potential genetic associations with ALS. This technology helps pinpoint genes that might not have been discovered otherwise. Such efforts push the boundaries of our understanding and open up numerous paths for researchers pursuing therapeutic interventions.
The progress in ALS therapeutic development is further accelerated by initiatives like the Target ALS In Vivo Target Validation studies, which play a pivotal role in bridging the gap between preclinical discoveries and clinical applications. By providing essential funding and resources, these studies enable researchers to validate promising therapeutic candidates in rigorous preclinical models. This critical step ensures that only the most viable treatments advance to clinical trials, expediting the development pipeline and bringing us closer to effective solutions for ALS.
Empowering Knowledge for ALS Awareness
ALS statistics paint a complex picture of a rare but devastating disease. As research advances, hope grows. New genetic insights and emerging therapies offer promise for better treatments. Armed with this knowledge, we’re better equipped to support those affected by ALS and advocate for continued research, so we can fulfill our vision for a world where Everyone with ALS Lives.
Frequently Asked Questions
What percentage of the population gets ALS?
ALS affects approximately 1 in 400 people, translating to about 0.33% of the population. It is more common in adults aged 40 to 70, with men being diagnosed slightly more often than women.
What is the life expectancy with ALS?
The average life expectancy for someone with ALS is typically 2 to 5 years after diagnosis, though some individuals may live 10 years or more. The progression of the disease can vary significantly based on factors like age, overall health, and the type of ALS.
Can ALS be stopped if caught early?
Currently, ALS cannot be stopped, even if caught early. However, early diagnosis can lead to better management of symptoms and improved quality of life.