Combined Gene Therapy Shows Promise, but Not Synergy, in Mice

A combined gene therapy that delivered two nerve growth factors — NRG1-I and NRG1-III — to muscle and nerve cells improved motor function and delayed disease onset in a mouse model of amyotrophic lateral sclerosis (ALS), a study demonstrated. However, the combo therapy did not show a synergistic effect,…

I spent most of my life following daily habits and routines that shaped my perspective about how life ought to be. When ALS appeared, it brought change and created imaginary roadblocks in my mind. I didn’t want things to change, to learn new habits, or to adapt to my…

The U.S. Food and Drug Administration (FDA) has cleared AB Science to resume patient enrollment in its confirmatory Phase 3 trial investigating masitinib as an add-on treatment in people with amyotrophic lateral sclerosis (ALS). AB Science put a temporary hold on all its masitinib clinical trials due…

The loss of chemical modifications in the regulatory sequence of the TARDBP gene, called DNA methylation, seems to contribute to increased levels and clumping of the TDP-43 protein in the motor cortex of people with amyotrophic lateral sclerosis (ALS), a study found. As methylation in this gene — which…

“You hold the key to love and fear All in your trembling hand Just one key unlocks them both It’s there at your command Come on people now Smile on your brother Everybody get together…

A Phase 2 clinical trial evaluating AL-S Pharma AG’s experimental therapy AP-101 in people with amyotrophic lateral sclerosis (ALS) has enrolled its first patient. The study (NCT05039099) already is recruiting up to 63 adults with sporadic and SOD1-related familial ALS at one of its Canadian sites.

Recently, a journalist and author from New York contacted me about a book he’s working on. His theme is the role that humor plays in helping people cope with a wide variety of diseases, disabilities, and physical conditions. He wanted to include my ALS-related experiences, and I gladly agreed to…

An abnormal version of tau, a protein associated with Alzheimer’s disease, may contribute to the progression of amyotrophic lateral sclerosis (ALS) by damaging mitochondria in neurons, according to a new study. The findings suggest that decreasing tau levels might be a new therapeutic strategy to lessen mitochondrial dysfunction in…

BioArctic AB announced that it is advancing work into selective antibodies against TAR DNA-binding protein 43 (TDP-43) that when misfolded can form the toxic clumps, or aggregates, believed to be involved in the development of amyotrophic lateral sclerosis (ALS) and other neurodegenerative disorders. One potential therapy approach is called…

Prilenia Therapeutics has raised $43 million to support potential regulatory submissions and marketing of its lead candidate, pridopidine, for the treatment of amyotrophic lateral sclerosis (ALS). The funding, obtained through a Series B financing round, also will be used to support the potential marketing of the candidate therapy for…