What is Dementia? Dementia is the persistent disorder of mental processes marked by difficulties with memory, changes in personality, and impaired reasoning. Dementia is not a specific disease, but rather an all-encompassing term for these cognitive impairments.  The cause of dementia varies and it can affect people differently by causing different symptoms. All patients suffering from dementia have experienced some damage to or loss of nerve cells and their connections in the brain.2 What are the Most Prevalent Types of Dementia? The different types of dementia are determined by the area ..read more

For over a decade, histone deacetylase (HDAC) inhibitors have been investigated as anti-cancer agents that play a role in the death or cell cycle arrest of cancer cells. But more recently, researchers have found increasing evidence that HDAC inhibition can alleviate symptoms of ALS. This is particularly the case for histone deacetylase 6 (HDAC6), thereby making it a target for potential gene therapies.   While 90% of ALS cases are sporadic, many of the remaining 10% of familial cases are related in some way to mutations in a handful of specific genes, including: SOD1 TDP-43 FUS C9orf72 ..read more

While the precise mechanisms that lead to the development of amyotrophic lateral sclerosis (ALS) are still under investigation, several genetic mutations have been linked to the disease and its symptoms. By investigating these mutations, researchers hope to discover therapeutic interventions that can slow down or stop the neurodegenerative effects of ALS.  Recently, researchers have found possible connections between hundreds of genes and ALS. Many of which may provide targets for new treatments.1 But there are a handful of well-researched genes that researchers believe mut ..read more

While the precise mechanisms that lead to the development of amyotrophic lateral sclerosis (ALS) are still under investigation, several genetic mutations have been linked to the disease and its symptoms. By investigating these mutations, researchers hope to discover therapeutic interventions that can slow down or stop the neurodegenerative effects  of ALS.  Recently, researchers have found possible connections between hundreds of genes and ALS. Many of which may provide targets for new treatments.1 But there are a handful of well-researched genes that researchers believe mu ..read more

Epidemiology is the method used to study health outcomes and the frequency of diseases in different groups of people. It’s crucial for researchers to understand the epidemiology, and especially the distribution, of amyotrophic lateral sclerosis (ALS) throughout micro and macro populations. Understanding epidemiological factors and collecting real-world data allow scientists worldwide to share information, compare and contrast risk factors in different populations, and collaborate on treatments. Collecting this valuable information can also help patients and families learn about the current sta ..read more

Gene therapy is promising biotechnology with the potential to treat or cure diseases caused by a mutated gene. By introducing new genetic material into a person’s cells, researchers hope to replace or repair faulty genes so that they can make beneficial proteins instead of toxic ones. The challenge in gene therapy is finding a way to get new genetic material inside the body and deliver it to human cells. Once researchers realized that viruses have the ability to deposit new DNA into cells, they found a way to harness that power. By removing the viruses’ damaging genetic material from cells and ..read more

Juvenile Amyotrophic Lateral Sclerosis (JALS), in which symptoms begin to appear before the age of 25, affects fewer than 1,000 people in the U.S.1 The overall prevalence of JALS worldwide is unknown.2 Perhaps the most famous case of the disease was physicist Stephen Hawking. He was diagnosed with juvenile ALS when he was 21 years old and lived with the disease for 55 years before passing away at age 76 (a very rare outcome).    The U.S. National ALS Registry indicates that the average age of disease onset is roughly 54 years, with only around 13% of patients developing symptoms ..read more

Neuroscientists are constantly researching to discover how best to diagnose ALS early in at-risk individuals and to improve the care of those living with the condition. Most ALS research studies typically focus on identifying the cellular and genetic mechanisms responsible for the development and progression of the disease.  There seem to be inadequate studies on ALS biomarkers, however, which are critical to enhancing early diagnosis, tracking disease progression, and clinical testing of potentially effective therapeutics for ALS.1 What Are Biomarkers? Biomarkers are measurable character ..read more

Neuroscientists are constantly researching to discover how best to diagnose ALS early in at-risk individuals and to improve the care of those living with the condition. Most ALS research studies typically focus on identifying the cellular and genetic mechanisms responsible for the development and progression of the disease.  There seem to be inadequate studies on ALS biomarkers, however, which are critical to enhancing early diagnosis, tracking disease progression, and clinical testing of potentially effective therapeutics for ALS.1 What Are Biomarkers? Biomarkers are measurable character ..read more

Currently, there is no single diagnostic test to confirm or rule out amyotrophic lateral sclerosis (ALS) in the earliest stages of the disease.1 Doctors must first rule out other conditions with similar symptoms and test for neurological symptoms to confirm an ALS diagnosis. Research is underway to develop a diagnostic test to identify ALS earlier, so interventions begin earlier.  Promising new research shows a muscle biopsy could potentially identify at-risk patients earlier in their disease. What is a Muscle Biopsy? Muscle biopsies,2  are frequently performed to determine if a pati ..read more