Acoramidis
Early-Stage Variant Transthyretin Amyloidosis (ATTRv)
- Preclinical
- Phase 1
- Phase 2
- Phase 3
- Approved
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Early-Stage Variant Transthyretin Amyloidosis (ATTRv)
Limb-Girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9)
Autosomal Dominant Hypocalcemia Type 1 (ADH1)
Chronic Hypoparathyroidism-NIH IIR
Achondroplasia
Hypochondroplasia
Canavan Disease
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