Acoramidis (AG10) – TTR stabilizer for transthyretin amyloidosis (ATTR)

ISA 2024 – Early increase in serum transthyretin level is an independent predictor of improved survival in ATTR cardiomyopathy

ISA 2024 – Acoramidis treatment-related increase in serum TTR is associated with lower cardiovascular mortality in ATTR-CM

ISA 2024 – Acoramidis treatment-related increase in serum TTR is associated with a lower risk of cardiovascular hospitalization in ATTR-CM patients

ISA 2024 – Acoramidis achieves early reduction in cardiovascular death or hospitalization in transthyretin amyloid cardiomyopathy (ATTR-CM)

ISA 2024 – Higher risk of mortality in previously hospitalized patients

ISA 2024 – Rationale & design of ACT-EARLY

ESC Heart Failure 2024 – ITT Sensitivity Analysis and Sub-Analysis Comparing Acoramidis and Placebo in Stage 4 CKD in ATTRibute-CM

ESC Heart Failure 2024 – NT-proBNP Insights from the ATTRibute-CM Study

ESC Heart Failure 2024 – EQ-5D Analysis from the ATTRibute-CM Study

ESC Heart Failure 2024 – Improved Health-Related Quality of Life in Acoramidis-Treated Patients with ATTR-CM, Demonstrated by Improvements in KCCQ Scores

ACC 2024 – CMR Imaging Substudy from ATTRibute-CM Phase 3 Study

The New England Journal of Medicine, Efficacy and Safety of Acoramidis in Transthyretin Amyloid Cardiomyopathy

ESC 2023 – Acoramidis Produces Near-Complete TTR Stabilization in Blood Samples from Patients with Variant Transthyretin Amyloidosis

Orphanet Journal of Rare Diseases, Patient and Family Experience with Transthyretin Amyloid Cardiomyopathy (ATTR-CM) and Polyneuropathy (ATTR-PN) Amyloidosis: Results of Two Focus Groups

Journal of the American College of Cardiology, Transthyretin Stabilization by AG10 in Symptomatic Transthyretin Amyloid Cardiomyopathy

Low-dose infigratinib – FGFR1-3 inhibitor for achondroplasia, hypochondroplasia & other skeletal dysplasias

Therapeutic Advances in Muscoskeletal Disease, Infigratinib in Children with Achondroplasia: The PROPEL and PROPEL 2 Studies

Nature Reviews: Endocrinology, International Consensus Statement on the Diagnosis, Multidisciplinary Management and Lifelong Care of Individuals with Achondroplasia

Bone, Lifetime Impact of Achondroplasia: Current Evidence and Perspectives on the Natural History

Genetics in Medicine, Achondroplasia Natural History Study (CLARITY): A Multicenter Retrospective Cohort Study of Achondroplasia in the United States

Orphanet Journal of Rare Diseases, Achondroplasia: A Comprehensive Clinical Review

The Journal of Clinical Investigation, Tyrosine Kinase Inhibitor NVP-BGJ398 Functionally Improves FGFR3-related Dwarfism in Mouse Model

Developmental Dynamics, Achondroplasia: Development, Pathogenesis, and Therapy

Encaleret – Ca sensing receptor antagonist for autosomal dominant hypocalcemia type 1 (ADH1)

The New England Journal of Medicine, Efficacy and Safety of Encaleret in Autosomal Dominant Hypocalcemia Type 1

Journal of Bone and Mineral Research, Autosomal Dominant Hypocalcemia Type 1: A Systematic Review

BBP-418 – Glycosylation substrate pro-drug for limb-girdle muscular dystrophy 2I/R9 (LGMD2I/R9)

Journal of Muscle Research and Cell Motility, Validation of a Novel Western Blot Assay to Monitor Patterns and Levels of Alpha Dystroglycan in Skeletal Muscle of Patients with Limb-girdle Muscular Dystrophies

PLOS One, Ribitol Dose-dependently Enhances Matriglycan Expression and Improves Muscle Function with Prolonged Life Span in Limb-girdle Muscular Dystrophy 2I/R9 Mouse Model

Nature Communications, Ribitol Restores Functionally Glycosylated Alpha-dystroglycan and Improves Muscle Function in Dystrophic FKRP-mutant Mice

BBP-812 – AAV9 gene therapy for Canavan disease

Neurology for Science 2023 – CANaspire Phase 1/2 Study

ASGCT 2023 – CANaspire Initial Biomarker and Clinical Findings

ASGCT 2021 – NHP