Reduced activity of the TDP-43 protein, a hallmark of amyotrophic lateral sclerosis (ALS), leads to changes in the DNA of nerve cells, which alters the activity of important genes, a new study reports. These findings may help explain how problems with this protein can contribute to the death of…
TDP-43 deficiency leads to changes in physical structures of cell DNA
A European Medicines Agency (EMA) committee again has delayed issuing an opinion on whether to grant conditional approval to masitinib, a potential add-on oral therapy for amyotrophic lateral sclerosis (ALS). AB Science, the therapy’s developer, now expects a recommendation in the second quarter of this year. The…
An early-stage clinical trial evaluating CK0803, Cellenkos‘ regulatory T-cell-based therapy for people with amyotrophic lateral sclerosis (ALS), has completed dosing its first patient group, the company announced in a press release. The six patients, all adults, were treated at the Columbia University Irving Medical Center, in New…
On the heels of promising HEALEY-ALS Phase 2 trial data, Prilenia Therapeutics plans to launch a pivotal Phase 3 study of pridopidine, its candidate amyotrophic lateral sclerosis (ALS) treatment, in the second half of this year. Relative to a placebo, pridopidine was associated with slower disease progression,…
Dewpoint Therapeutics has received a grant from the Target ALS Foundation to advance the development of small molecules called c-mods, which will target the TDP-43 condensates thought to drive most cases of amyotrophic lateral sclerosis (ALS). Condensates are borderless compartments in cells that can cause cellular processes…
As my husband Todd’s ALS progressed, trips to the doctor’s office became increasingly difficult. When he was still walking, we used to travel four hours from northern Michigan through Wisconsin to the closest ALS clinic in Duluth, Minnesota. We turned those trips into mini-vacations, bringing the kids along and…
Blocking certain inflammatory signaling pathways may be one way to reverse nerve cell alterations that drive the symptoms of amyotrophic lateral sclerosis (ALS), according to preclinical research using a mouse model of the disease. In the mice, an ALS-associated mutation in the FUS gene caused caused motor neurons — the…
Up to three years of daily treatment with the investigational therapy IPL344 was well tolerated and slowed disease progression among adults with amyotrophic lateral sclerosis (ALS), according to top-line data from a small Phase 1/2a clinical trial. Preliminary efficacy analyses also indicated that IPL344 might be able to…
Living with ALS for the past 14 years has taught me an important lesson on adapting to a new way of life. Before ALS, my life was filled with carefree movement. I spoke with ease and enjoyed endless physical energy. Now, even though my…
Note: This story was updated Jan 24, 2024, to correct that Radicava and Radicava Oral Suspension are available in Switzerland but not in other European countries. FAB122, an oral formulation of edaravone developed by Ferrer, failed to slow disease progression or extend survival in adults with amyotrophic lateral…
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