Tag: ALS

March 12, 2024

For Rick Bedlack, director of the ALS clinic at Duke University, tough times come with the job. He and his team treat about 500 people with the rare, fatal disorder that rapidly erodes nerve cells. Friday, though, was particularly difficult. […]

March 11, 2024

Following disappointing trial results, the maker of a controversial ALS drug may pull the medication off the market. In a statement issued Friday, Amylyx Pharmaceuticals said that Relyvrio failed to help patients in a large follow-up study, but the company […]

June 6, 2023

The Food and Drug Administration has set a date for an upcoming meeting in which an outside group of experts will evaluate an experimental ALS medicine the agency has appeared wary of approving. The medicine’s developer, Brainstorm Cell Therapeutics, disclosed […]

April 26, 2023

The U.S. Food and Drug Administration on Tuesday approved a new drug for people who have a rare, inherited type of amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease. The medication, toferson (Qalsody), targets a mutation in the […]

March 27, 2023

A yearslong disagreement between the Food and Drug Administration and a developer of an experimental medicine for ALS will get a public hearing sometime in the future, setting up another high-profile debate of what amount of evidence merits an approval […]

January 31, 2023

Apic Bio, a privately held developer of gene therapies for rare disorders, has agreed to sell the rights to one of its experimental ALS treatments to the biotechnology company UniQure in a deal announced Tuesday. Per deal terms, UniQure will […]

November 9, 2022

A pre-clinical pipeline of potential first-in-class brain-penetrant small molecule inhibitors of the mitochondrial permeability pore (mPTP) are to be developed. These will act through a novel mechanism of action and be used in treatment of Parkinson’s and Amyotrophic Lateral Sclerosis […]

September 22, 2022

In an open-label extension of a Phase III clinical trial sponsored by the pharmaceutical company Biogen, investigators have shown that the use of the drug tofersen for one year may help stabilize muscle strength and motor control in patients with […]

September 6, 2022

U.S. Food and Drug Administration panel will once again consider approval for an experimental drug for ALS, a rare second review for a disease that has no cure. The same panel that will meet Wednesday voted last March not to […]

September 5, 2022

Cedars-Sinai investigators have developed an investigational therapy using support cells and a protective protein that can be delivered past the blood-brain barrier. This combined stem cell and gene therapy can potentially protect diseased motor neurons in the spinal cord of […]

May 13, 2022

Mitsubishi Tanabe Pharma Corporation (MTPA) today announced the U.S. Food and Drug Administration (FDA) has approved Radicava ORS (edaravone), the oral form of edaravone, for the treatment of amyotrophic lateral sclerosis (ALS), a neurodegenerative disease that currently has no cure […]

October 18, 2021

Stop me if you think you’ve heard this one before. A Biogen neurodegenerative disease prospect has failed a pivotal study, but the Big Biotech has latched onto biomarker data and “trends favoring” the treatment as it heads into talks with […]

September 28, 2021

Myeloperoxidase (MPO) inhibitor verdiperstat was no better than placebo in the M-STAR trial in patients with MSA, and looks likely to be abandoned for this indication. It’s not the end of the line for the drug, which Biohaven picked up […]