Combination therapies are generating some interest now, for example pairing ASO with muscle-protecting agents or anti-inflammatory compounds to enhance functional outcomes. Genome-editing technologies as CRISPR-Cas9 offer probably the potential for more permanent correction of pathogenic mutations in the next future. We’re also seeing progress in biomarkers, in developing biomarkers...
Combination therapies are generating some interest now, for example pairing ASO with muscle-protecting agents or anti-inflammatory compounds to enhance functional outcomes. Genome-editing technologies as CRISPR-Cas9 offer probably the potential for more permanent correction of pathogenic mutations in the next future. We’re also seeing progress in biomarkers, in developing biomarkers. This is important for early diagnosis. We have to treat patients early, so we need early diagnosis. So the development of novel biomarkers is crucial for this process and for monitoring also the therapeutic response, the therapeutic effects. Lastly, patient-specific iPSC models at this moment are under study and are enhancing our ability to study disease mechanisms and predict treatment efficacy so there are many points to study in the next future in our field. I believe we’ll see broader applications of personalized medicine supported by multiomic profiling and probably AI-driven data integration. Future therapies will likely be combination-based and tailored not just by genotype but by disease stage individual response profiles. Gene editing, I already said, may go from preclinical to clinical practice in select neuromuscular conditions. I think with increasing collaboration with the university, academia, biotech, and regulatory bodies think that the pace of innovation will only accelerate in the next future. It’s important to have a good understanding of genetics of molecular biology and how these apply to clinical practice. Stay updated with the new treatments and guidelines of course. Also develop communication skills to explain the complex therapies to patients and families. And finally I believe remember that interdisciplinary care is a key point neurologists, geneticists, epidemiologists, physical therapist, and other doctors all play a role in managing these conditions and increasing the quality of life of our patients.
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