Author Archives: Joana Fernandes, PhD

Increasing the production of the muscle protein Dok-7 may be a way to treat the muscle weakness in ALS, a Japanese study reports. The research, “DOK7 genes therapy enhances motor activity and life span in ALS model mice,” was published in the journal EMBO Molecular Medicine. A hallmark…

Flex Pharma is launching the Phase 2 COMMEND trial, which will investigate the effects of FLX-787 in patients with muscle cramps associated with amyotrophic lateral sclerosis (ALS). The preliminary results of the trial are expected to be released in mid-2018. The COMMEND trial (NCT03196375) will start with an assessment…

Scientists have known for some time that abnormal DNA repetitions in the C9orf72 gene contribute to neuron death in ALS and frontotemporal dementia. Now they have learned how it happens: The anomalies leave DNA susceptible to damage, prompting a cell repair mechanism to become over-active. That hyperactivity cause neuron deaths.

Researchers have discovered a natural mechanism that can help prevent the formation of harmful protein clumps that contribute to the development of amyotrophic lateral sclerosis (ALS), according to results of a new study. They also gained new insights into the formation of the protein clumps known as TDP-43 protein aggregates that…

Massachusetts General Hospital in Boston and California Pacific Medical Center in San Francisco will be among the sites where ALS patients will take in a Phase 3 clinical trial of BrainStorm Cell Therapeutics’ NurOwn. The trial will begin after the company receives approval from the U.S. Food and Drug Administration and…

A musical instrument that can be played only with brain activity may empower and rehabilitate patients with ALS and other motor diseases. Invoking brain scans known as electroencephalograms, University of Washington researchers have dubbed the musical device they created an Encephalophone. The article they wrote about it, titled “A Novel Musical…

Neuraltus Pharmaceuticals has completed enrollment its Phase 2 confirmatory study of NP001 (NCT02794857) in ALS patients with systemic inflammation. The company expects the last patient in the trial to be assessed in early 2018 and to release the study’s  results in the first months of the year. “Reaching…

A moderate to severe traumatic brain injury puts a person at high risk of developing dementia, although not Parkinson’s or ALS, a study in Finland suggests. In contrast, a mild brain injury appears to have no connection with the development of dementia, the researchers said. The study, which involved brain injuries…

Several metals may be linked to the risk of amyotrophic lateral sclerosis (ALS), according to results of a small study in Italy. The findings state that levels of metals and other essential trace elements in blood, urine, and hair may serve as biomarkers to diagnose and monitor ALS. The study titled,…

The former malaria therapy pyrimethamine reduces levels of an ALS-linked protein in the spinal fluid of patients whose disease stems from gene mutations, a Phase 1/2 clinical trial shows. The protein is SOD1. The mutations connected with amyotrophic lateral sclerosis are in the SOD1 gene that controls the protein. The…