Retrotope’s Experimental RT001 Now Available for ALS Under Expanded Access Program

Retrotope is providing its investigational fatty acid being developed for Friedreich’s ataxia, RT001, to people with amyotrophic lateral sclerosis (ALS) under an expanded access program, also called “compassionate use.” Individual patients with life-threatening or severely debilitating diseases can petition the U.S. Food and Drug Administration, through their physicians,…

Depleting a protein called Staufen1 improved motor function and lowered protein aggregation in a mouse model of spinocerebellar ataxia type 2 (SCA2). The findings suggest that targeting Staufen1 could lead to therapies for amyotrophic lateral sclerosis (ALS) and other neurodegenerative disorders. The study “Staufen1 links…

“Manners are a sensitive awareness of the feelings of others. If you have that awareness, you have good manners, no matter what fork you use.” ―Emily Post In case we ever meet, I want to apologize in advance if I ever appear insensitive to, or unaware of, your feelings. That is…

If you’re like me and live with ongoing physical changes from ALS, you’ve probably used workarounds. What is a workaround? A workaround is a creative, temporary solution that solves an everyday problem. For example, in the real world, folks put red duct tape over a broken tail light…

A man with progressive non-fluent aphasia (PNFA), one of the three types of frontotemporal dementia (FTD), was unusual in developing amyotrophic lateral sclerosis (ALS) with bulbar-onset one year later, a case study from Italy reports. The study, “A case of Progressive Non-Fluent Aphasia as onset of Amyotrophic Lateral…

The U.S. Food and Drug Administration (FDA) has approved Tiglutik, an oral suspension of riluzole, for the treatment of amyotrophic lateral sclerosis (ALS). Rilutek (riluzole, by Sanofi) has been available in the U.S. as 50 mg tablets since December 1995. However, ALS patients can have difficulties with…