A committee of the European Medicines Agency (EMA) is leaning toward not recommending a conditional approval of Amylyx Pharmaceuticals’ AMX0035 for treating amyotrophic lateral sclerosis (ALS) in the European Union. The drug is already marketed in the U.S. under the name Relyvrio (sodium phenylbutyrate and taurursodiol) and…

WVE-004, Wave Life Sciences‘ investigational treatment for amyotrophic lateral sclerosis (ALS), significantly reduces toxic proteins associated with C9orf72 genetic mutations, but that doesn’t seem to translate into functional status gains in patients. Based on findings from the Phase 1b/2a FOCUS-C9 trial (NCT04931862), the company has decided to discontinue developing…

Mary Kate Karam and Brian Jeansonne at Jazz Fest 2023, watching Mumford & Sons, Trombone Shorty, and John Batiste. Incredibly hot, but incredibly fun! (Photo courtesy of Mary Kate Karam) This is Mary Kate Karam’s story: Brian Jeansonne, a father of five, was diagnosed…

“You should burn the beaver,” my husband, Todd, said. Over the last several years, since he has been paralyzed due to ALS, he has given me a lot of advice on various house projects, but that was not something I ever anticipated hearing. Last weekend, I was walking with…

Cognixion’s flagship device Cognixion ONE Axon, a product designed to improve communication in people with severe motor impairments, has beed granted breakthrough device designation by the U.S. Food and Drug Administration (FDA). The wireless device is specifically designed for people with people with amyotrophic lateral sclerosis (ALS)…

MRI scans of the brains of people with amyotrophic lateral sclerosis (ALS) have identified a neurochemical called N-acetyl aspartate (NAA) whose levels correlate with the loss of motor function, a study reports. A drop in NAA levels was associated with the breakdown in communication between the primary motor cortex…

May has always been my favorite month of the year. Here in Maryland, the trees have filled in with lush green leaves, commercial crabbing boats have begun to quietly travel my small creek in the early dawn hours, and mallard ducks have returned to my yard. The evenings stay lighter…

Daily oral treatment with memantine did not slow disease progression nor did it reduce biomarker changes among people with amyotrophic lateral sclerosis (ALS) enrolled in a Phase 2b trial. Approved under the brand name Namenda to treat cognitive problems in people with Alzheimer’s disease, memantine has also…

Treatment with the investigational therapy DNL343 was generally well-tolerated among people with amyotrophic lateral sclerosis (ALS) in an early clinical trial, and biomarker data from the trial suggest that the therapy is working as intended. Results were presented at the annual meeting of the American Academy of Neurology (AAN),…

An immune protein that is mostly produced in neurons was found to drive damage to mitochondria, which produce energy for cells, and nerve fiber loss, a study has found. Suppressing the protein, called gasdermin-E (GSDME), prevented nerve fiber loss in cells derived from people with amyotrophic lateral sclerosis (ALS),…