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,…
Retrotope’s Experimental RT001 Now Available for ALS Under Expanded Access Program
Human spinal cord neural stem cells (NSC), created using an innovative method, were seen to regenerate functional neurons in the damaged tissue of rats with spinal injuries, according to researchers. Their study, “Generation and post-injury integration of human spinal cord neural stem cells,” was published in the journal…
Amyotrophic lateral sclerosis’ toll on the thinking abilities and behavior of patients is as progressive and damaging as the disease’s physical toll, researchers in the U.K. report, with ALS-specific cognitive and behavioral deficits showing an impact even in early stages and worsening as the disease advances. ALS is characterized by the…
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…
Survival and quality of life of patients with more advanced amyotrophic lateral sclerosis (ALS) can be improved significantly with the use of noninvasive mechanical ventilation (NIV). But many factors can contribute to a delayed decision on when to start mechanical ventilation, as well as on the effectiveness of the…
The ALS Workaround Dilemma
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 new process by which pro-death signaling is sent to nerve cells may hold the key for treating neurodegenerative diseases such as amyotrophic lateral sclerosis (ALS), which is characterized by the loss of motor nerve cells, according to researchers. The study, “Retrograde degenerative signaling mediated by the p75…
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…
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