Ultragenyx Pharmaceutical (United States)

Recent papers from Ultragenyx Pharmaceutical (United States)

1. Health-Related Quality of Life in Homozygous Familial Hypercholesterolemia: Results from an Online SurveyPatient Preference and Adherence · 2026
2. Impact of Evinacumab on Coronary Plaques in Patients With Homozygous Familial Hypercholesterolemia: Protocol of the "Evolve-Hofh" StudyAtherosclerosis · 2026
3. Real-World Study on the Effectiveness of Standard of Care in Lowering LDL-C Levels for Primary Prevention of Atherosclerotic Cardiovascular Disease in JapanAtherosclerosis · 2026
4. Adverse Events in the Evinacumab Clinical Development Programme in Homozygous Familial Hypercholesterolaemia (Hofh): Focus on Immunogenicity, Liver Function, and PregnancyAtherosclerosis · 2026
5. Clinical Efficacy and Safety of Evinacumab Initiated Through a Global Compassionate Use Programme for Patients With Homozygous Familial Hypercholesterolaemia (Hofh)Atherosclerosis · 2026
6. Effect of Evinacumab on Lp(a) Levels in Adults and Adolescents With Homozygous Familial Hypercholesterolaemia (Hofh): Insights From the Elipse-Open Label Extension StudyAtherosclerosis · 2026
7. Development of an Interactive Database of Ldlr, Apob, Pcsk9, and Ldlrap1 Variants and Genotypes Observed in Homozygous Familial Hypercholesterolemia (Hofh)Atherosclerosis · 2026
8. Fractures in osteogenesis imperfecta: a review of the frequency, location, and impact on patient quality of lifeOrphanet Journal of Rare Diseases · 2026
9. Beyond Seizure Management in a Developmental Epileptic EncephalopathyNeurology Genetics · 2026
10. Phase 3 Randomized Trial Results of DTX401 AAV Gene Therapy for the Treatment of GSDIaJournal of Inherited Metabolic Disease · 2026
11. A matched, real-world observational study of the burden of osteogenesis imperfecta and healthcare resource utilization in Ontario, CanadaBMC Musculoskeletal Disorders · 2026
12. Characterization and Analysis of PHEX Variants in Patients With Hypophosphatemia in ArgentinaAmerican Journal of Medical Genetics Part A · 2026
13. Real-world outcomes in patients with tumor-induced osteomalacia treated vs not treated with burosumabJournal of the Endocrine Society · 2026
14. Cross-sectional MRI-based quantification of central nervous system tissue and cerebrospinal fluid volumes in juvenile domestic pigsFrontiers in Veterinary Science · 2026
15. A MOF-Based Strategy for Synthesizing High-Strength Single-Crystal NCM811 Cathode MaterialsEnergy & Fuels · 2026
16. A mechanistic model for recombinant adeno‐associated virus production dynamics in a helper virus‐assisted producer cell lineBiotechnology Progress · 2026
17. External controls for rare disease drug development: Lessons for emerging and advanced therapeutic modalitiesMolecular Therapy Advances · 2026
18. Healthcare resource use, treatment patterns, and costs for patients living with osteogenesis imperfecta—results from a US retrospective claims databaseFuture Rare Diseases · 2026
19. Real-world Effectiveness of Burosumab Across Age Groups: X-linked Hypophosphatemia (XLH) Disease Monitoring ProgramThe Journal of Clinical Endocrinology & Metabolism · 2026
20. Disulfide bond reduction of antibodies during clarification: A bench‐scale assay and a mitigation toolBiotechnology Progress · 2026