“Increasing Adoption of Artificial Intelligence (AI) and Automation Technologies”
One notable trend in Machado-Joseph Disease (MJD) treatment is the growing focus on gene therapy and protein degradation strategies to target the underlying genetic cause of the disease. Traditional treatments have mainly focused on symptom management, such as using L-dopa for movement disorders and botulinum toxin for spasticity. However, recent advancements have shifted toward therapies aimed at reducing the toxic protein accumulation caused by the mutation in the ATXN3 gene. For instance, SLS-005 (trehalose), a drug that has shown promise in clinical trials, works by enhancing the degradation of misfolded proteins, potentially slowing disease progression. Additionally, gene-editing technologies such as CRISPR-Cas9 are being explored as a future approach to correct the genetic mutation at its source. The increasing investment in these cutting-edge therapies offers hope for alleviating symptoms and potentially halting or reversing the course of MJD, providing a more targeted, long-term solution for patients.
