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PRESENTATION · Mar 2025 · PDF

Acoramidis TTR - ACC 2025 -Healthcare Disparities and Diagnostic Trends

PRESENTATION · Mar 2025 · PDF

BBP-418 - MDA 2025 - Understanding the Burden of LGMD2I/R9

PRESENTATION · Mar 2025 · PDF

BBP-418 - MDA 2025 - Baseline Characteristics FORTIFY

PUBLICATION · Mar 2025 · PDF

Journal of the American College of Cardiology, Efficacy of Acoramidis on All-Cause Mortality and Cardiovascular Hospitalization in Transthyretin Amyloid Cardiomyopathy

PUBLICATION · Mar 2025 · PDF

Circulation, Long-Term Efficacy and Safety of Acoramidis in ATTR-CM: Initial Report from the Open-Label Extension of the ATTRibute-CM Trial

PUBLICATION · Mar 2025

Pediatric Neurology, Feasibility, Acceptability, and Reliability of Remote Motor Assessment in Children with Canavan Disease

PUBLICATION · Jan 2025

Human Gene Therapy, Urine N-Acetylaspartate Distinguishes Phenotypes in Canavan Disease

PRESENTATION · Dec 2024 · PDF

Acoramidis - AHA 2024 - Acoramidis reduces ACM and CVH: initial outcomes from the ATTRibute-CM open label extension study

PRESENTATION · Dec 2024 · PDF

Infigratinib - ISDS 2024 - Clinical Development Program Update

PRESENTATION · Dec 2024 · PDF

Encaleret - ADH1 - ASBMR 2024 Phase 2B Data over 24 Months

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Breaking news for the achondroplasia community

Positive Topline Phase 3 Data from PROPEL 3, our Study of Oral Infigratinib
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