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Next-generation sequencing (NGS) allows for efficient analysis of known genetic mutations, enhancing diagnostic accuracy. Overall, the diagnostic process for LOPD typically involves a series of assessments, including clinical, laboratory and imaging tests, culminating in geneticanalysis to confirm the diagnosis.
As of January 31, 2024, approximately 131 unique RNA-based therapies are in clinicaldevelopment across various therapeutic areas. While these therapies span all stages of clinicaldevelopment, they are particularly concentrated in earlier phases, indicating strong future growth. billion in 2022 to $27.0
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