PTC Therapeutics (United States)

Recent papers from PTC Therapeutics (United States)

1. Moisture-path-dependent thermal resistivity hysteresis of compacted soils for energy and geotechnical systemsScientific Reports · 2026
2. MAPT splicing modulators reduce 4R tau and rescue tauopathy phenotypes in human neurons and in a mouse modelScience Translational Medicine · 2026
3. Regulatory harmonization: Evolution, globalization and future directionsBritish Journal of Clinical Pharmacology · 2026
4. Utility of clinical scoring systems, laboratory and genetic testing for appropriate classification of severe Hypertriglyceridemic patients in a Latin American CohortJournal of clinical lipidology · 2026
5. Novel system for rapid soil thermal conductivity with constrained environmental conditionsDiscover Civil Engineering · 2026
6. Ethnic Bridging of Votoplam, a HTT Gene Splicing Modifier, Using Pharmacokinetic Comparison Between Caucasian and Japanese Subjects.2026
7. Ethnic Bridging of Sepiapterin in Chinese and Korean Populations Based on Predictions From Genetic Polymorphism of Breast Cancer Resistance ProteinClinical and Translational Science · 2026
8. Abstract 2552: Cell-free RNA urogentital cancer biomarker detection from urineCancer Research · 2026
9. Abstract 5381: Integrated analysis for identification of risk stratification biomarkers for colon cancer.Cancer Research · 2026
10. Efficacy and safety of sepiapterin versus sapropterin in patients with phenylketonuria: Results from the Phase 3, randomized, crossover, open-label, active-controlled AMPLIPHY trialMetabolism · 2026
11. Pharmacodynamics, Efficacy, and Safety of Intraputaminal Eladocagene Exuparvovec Administered to Pediatric Patients With Aromatic L‐Amino Acid Decarboxylase Deficiency Using an MR ‐Compatible Cannula: 48 Weeks of Follow‐UpJournal of Inherited Metabolic Disease · 2026
12. No QTcF Prolongation with Sepiapterin: Results From a Thorough QT Study in Healthy Subjects at Therapeutic and Supratherapeutic DosesThe Journal of Clinical Pharmacology · 2026
13. Effect of long-term sepiapterin treatment on dietary phenylalanine tolerance in patients with phenylketonuria: Interim results from the phase 3 APHENITY Extension StudyGenetics in Medicine · 2026
14. Exploratory Analysis of ELP1 Expression in Whole Blood From Patients With Familial DysautonomiaAnnals of Clinical and Translational Neurology · 2025
15. Amyotrophic lateral sclerosis in Colombia: a population-based study of incidence and socioeconomic determinantsAmyotrophic Lateral Sclerosis and Frontotemporal Degeneration · 2025
16. Effects of High‐ and Low‐Fat Meals on the Bioavailability and Pharmacokinetics of Votoplam, a HTT Gene Splicing ModifierClinical Pharmacology in Drug Development · 2025
17. Leveraging Medulloblastoma Clonal Dynamics to Overcome Treatment ResistanceClinical Cancer Research · 2025
18. Effectiveness of Ataluren in Patients with nmDMD: Confirmatory Evidence from the STRIDE RegistryNeuropediatrics · 2025
19. Eladocagene Exuparvovec for the Treatment of Aromatic l-Amino Acid Decarboxylase Deficiency (AADCd): An Economic Evaluation from a US PerspectivePharmacoEconomics · 2025
20. Enhanced Vatiquinone Bioavailability With Fatty Meals and Optimal Dosing ScheduleBiopharmaceutics & Drug Disposition · 2025