The U.S. Food and Drug Administration has granted fast track status to FLX-787, Flex Pharma‘s treatment for severe muscle cramps in amyotrophic lateral sclerosis. Flex designed FLX-787 to interfere with transient receptor potential ion channels that are believed to play a role in nervous system pain and inflammation.
News
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.
Brainstorm Gets $16 Million California Grant for Trial of NurOwn, Its Stem Cell Therapy for ALS
Brainstorm Cell Therapeutics has received a $16 million grant from a state agency known as the California Institute for Regenerative Medicine to develop a Phase 3 clinical trial of its ALS stem-cell therapy NurOwn. NurOwn is based on mesenchymal stem cells, which can transform themselves into various types of cells. Brainstorm…
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…
RNA molecules that are not supposed to leave the cell nucleus might be responsible for driving neuronal cell death in people with amyotrophic lateral sclerosis (ALS), researchers from England’s University of Sheffield report in a study that suggests a way to prevent that from happening. The research team discovered just how…
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…
Although known risks factors for ALS include age, male sex, and cigarette smoking, how occupations can influence ALS or other neurodegenerative diseases is less understood. A new study, however, finds that those who have high-status careers, often called white collar workers, face higher Amyotrophic lateral sclerosis (ALS) and Parkinson’s disease (PD)…
The Orlando-area medical marijuana company Green Relief is reminding Florida residents that the state no longer requires a 90-day waiting period before a patient with a serious disease like ALS can receive treatment. Florida lifted the 90-day requirement June 24 with rules that implemented changes in the state’s medical marijuana law…
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