Disease-modifying therapies

Amyotrophic lateral sclerosis (ALS) is a lethal neurodegenerative disease that lacks effective therapeutic strategies to date. GemPharmatech (GPT) generated the GPT hSOD1 G93A transgenic mouse strain to recapitulate ALS-like phenotypes in an animal model, in support of medical research and drug development. The hSOD1 G93A mouse model carries the G93A mutation in human SOD1 gene, which is widely expressed in mice. We observed an apparent body weight decrease in 25-week-old mice. We further performed a range of behavioral tests to identify the disease onset timing in GPT hSOD1 G93A mouse model,

Current disease-modifying therapies in multiple sclerosis are all focused on suppressing the inflammatory phase of the disease. This has been extremely successful, and it is doubtful that significantly more efficacious anti-inflammatory treatments will be found. However, it remains the case that people with relapsing-remitting multiple sclerosis acquire disability on treatment, and enter the secondary progressive phase. I argue that we now need treatments that prevent neuronal degeneration. The most promising approach is to prevent axons degenerating by remyelination. Since the discovery that

…use in developing neuroprotective disease-modifying therapies (DMTs), because DMTs require interventions at much earlier stages of PD when vulnerable neurons are still intact. Because PD patients exhibit various non-motor prodromal symptoms (ie, symptoms that predate diagnosis), understanding the pathophysiology underlying these symptom could lead

There are no effective disease-modifying therapies for neurodegenerative diseases such as Alzheimer’s, Parkinson’s, amyotrophic lateral sclerosis or Huntington’s disease. Huntington’s disease (HD) is a devastating autosomal dominantly inherited neurodegenerative disease and the world’s most common genetic dementia. I will present an overview of important approaches in development for targeting mutant HTT DNA and RNA (Tabrizi et al Neuron 2019), the cause of HD pathogenesis, and the translational pathway from bench to clinic for a HTT targeting antisense oligonucleotide (Tabrizi et al New Engla