The SMA Market: Assessing The Unknowns
Treatment Progress Leads To New Challenges In Spinal Muscular Atrophy
The introductions of Spinraza and Zolgensma in SMA offer new insights into how to address neurodegenerative diseases. But more real world evidence is needed.
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The introduction of gene therapies has been the highest-profile new modality to reach the market, but new RNA-based therapeutics hold significant potential and could be more accessible for patients. Scrip reviews the leading drug developers in this space.
High price tag, thin evidence of clinical efficacy for Exondys 51 result in low score from ICER, but a re-do is almost certain when confirmatory data on eteplirsen are released.