The U.S. Food and Drug Administration (FDA) has accepted for review the new drug application (NDA) of Biohaven Pharmaceuticals’ BHV-0223, a sublingual (dissolved under the tongue) form of riluzole, as a treatment for amyotrophic lateral sclerosis (ALS). Biohaven has begun enrollment of ALS patients for its Phase 2/3…
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Amyotrophic lateral sclerosis (ALS) or frontotemporal lobar degeneration (FTLD) patients with the C9ORF72 mutation are at an unusually high risk of malignant skin cancer and should be monitored for it, a study suggests. The study, “Increased risk of melanoma in C9ORF72…
Loss of C9orf72, a protein whose corresponding gene is among those most often mutated in amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD), causes an imbalance in the metabolism of fats (lipids) in motor neurons, a new study shows. These findings suggest that an imbalance in energy and lipid metabolism…
Specific proteins analyzed in cerebral spinal fluid and blood samples may help to diagnose amyotrophic lateral sclerosis (ALS) and predict patient outcomes, researchers report. Cerebral spinal fluid (CFS) protein patterns helped to identify 94% of ALS patients in their study, and the levels of three specific proteins were seen to…
Amyotrophic lateral sclerosis (ALS) is a condition that is challenging to diagnose because it shares many common symptoms with other diseases. Blood tests are used to look for evidence of diseases, the symptoms of which are similar to those of ALS. They can help, therefore, to…
Amyotrophic lateral sclerosis (ALS), a progressive neurological disorder, can be challenging to diagnose because its obvious symptoms are common to other neurological conditions. A thyroid function test can help in a search for evidence of other conditions with shared symptoms and to rule out ALS, which is not associated with…
The prevalence of motor nerve diseases — including amyotrophic lateral sclerosis (ALS) — is on the rise worldwide, mostly because of an aging population. The disease burden is greatest in high-income countries, according to a global analysis of data from 1990 to 2016. The study, “Global, regional,…
To bring attention to the plight of teens touched by amyotrophic lateral sclerosis (ALS), Mitsubishi Tanabe Pharma America (MTPA) is calling for artistic submissions from young people who have a family member with the disease. Called ALSO US, the new initiative is open to U.S. teenagers and young…
Mutations in a gene linked to familial amyotrophic lateral sclerosis (ALS), called FUS, affect the ability of motor nerve cells to repair damage to their DNA, a new study shows. These findings suggest that therapies that enhance the repair of DNA damage may be of benefit to some ALS patients.
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Multiple Rare Mutations in Genes Linked to ALS Can Lead to Earlier Disease Onset, Study Reports
People with multiple rare mutations in genes known to cause amyotrophic lateral sclerosis (ALS) are significantly more likely to develop this disease at younger ages, a study in Japanese patients reports. The study, “Burden of rare variants in causative genes for amyotrophic lateral sclerosis (ALS) accelerates…