Cytokinetics has completed patient enrollment in its Phase 2 trial to evaluate the safety and potential of reldesemtiv to improve muscle function in patients with amyotrophic lateral sclerosis (ALS), the company announced. The trial (NCT03160898), called FORTITUDE-ALS, includes about 450 ALS patients in the U.S., Canada, Europe,…
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Amyotrophic lateral sclerosis (ALS) is a progressive neurological condition that affects motor neurons. Motor neurons are nerve cells that control the movement of voluntary muscles, including respiratory muscles. Respiratory failure is the most common cause of death in ALS patients. Lung function tests are used to assess the risk…
Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease, and inheritance is thought to play an essential role in its less common form, called familial ALS (FALS). A genetic test is usually only performed on individuals with a known family history of ALS. Familial versus sporadic ALS The…
NSI-566 is an experimental stem cell-based therapy that Seneca Biopharma (previously known as Neuralstem) is developing as a potential treatment for amyotrophic lateral sclerosis (ALS). The company also is investigating NSI-566 as a potential treatment for chronic spinal cord injury and to restore motor deficits…
Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease that affects the nerves that control muscle function. The disease leads to muscle weakness, including the muscles needed for breathing. This can be very serious, with respiratory failure being the primary cause of death in ALS patients. Supporting breathing…
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…
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…