Engensis Gene Therapy Found Safe in Small Phase 2a Trial

Engensis Gene Therapy Found Safe in Small Phase 2a Trial

Repeated muscle injections with Engensis (VM202), Helixmith’s investigational non-viral gene therapy, were generally safe and well-tolerated in people with amyotrophic lateral sclerosis (ALS), according to top-line data from a Phase 2a clinical trial. While the sample size was too small to determine the therapy’s efficacy, muscle biopsies were…

Cholesterol Transporter May Be Strongest Trigger of Sporadic ALS

The cerebrospinal fluid of people with sporadic amyotrophic lateral sclerosis (sALS), but not of those with familial forms of the disease, promoted ALS-specific molecular, cellular, and motor abnormalities in healthy mice, a study showed. Apolipoprotein B-100 (apoB100), a protein involved in cholesterol transport, was identified as the main…

How ALS Is Teaching Me to Start Over — Again and Again

I’ve been living with ALS for 12 years now, and it’s certainly taught me many lessons. They’ve involved having patience and perseverance, as well as getting back up over and over. The most important lesson, however, has been to let the present moment be what it is, instead of…