Intellia Therapeutics Receives U.S. FDA Orphan Drug Designation for NTLA-2001, an Investigational CRISPR Therapy for the Treatment of Transthyretin (ATTR) Amyloidosis

NTLA-2001 has the potential to be the first single-dose treatment for ATTR … which can lead to nerve damage, or cardiomyopathy (ATTRv-CM), …
Source: nerve pain medication

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