The rare neurology and neuromuscular treatment landscape is rapidly transforming with the emergence of innovative disease-modifying technologies, such as gene therapy and RNA-based therapies. Our team is tracking several factors in this therapy area that we expect to significantly influence treatment and market dynamics including:
- The shortage of neurologists
- Continued drug development focus on gene and antisense oligonucleotide therapies
- Regulatory guidance and accelerated drug approvals based on biomarkers in slowly progressive neurological diseases (e.g., Duchenne Muscular Dystrophy, Alzheimer’s disease)
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