Rare Disease Therapeutics (United States)

Recent papers from Rare Disease Therapeutics (United States)

1. Deramiocel heart-derived cellular therapy in advanced Duchenne muscular dystrophy (HOPE-3): a phase 3, randomised, double-blind, placebo-controlled trialThe Lancet · 2026
2. Use of SGLT2 Inhibitors in Dystrophin Deficient Cardiomyopathy: A Multi-center Study Using the ACTION RegistryPediatric Cardiology · 2026
3. Effects of sevasemten (EDG-5506) on safety, biomarkers, and functional measures in adults with Becker muscular dystrophy: results of a phase 1b, open-label studyEBioMedicine · 2026
4. Deramiocel Significantly Slows Upper Limb Functional Decline in Duchenne Muscular Dystrophy: Skeletal Muscle Outcomes from the Phase 3 HOPE-3 Trial (LS2.003)Neurology · 2026
5. Closing the Gap to Interventions for Tuberous Sclerosis Complex–Associated Neuropsychiatric Disorders (TAND): Protocol for a Longitudinal Study of TAND Severity, Predictors, and Caregiver Well-Being (TANDem-2)JMIR Research Protocols · 2026
6. Heart Transplantation and Ventricular Assist Device in Duchenne Muscular Dystrophy: A New EraPediatric Transplantation · 2025
7. Exposure-response of serum biomarkers to vamorolone, a dissociative corticosteroidal anti-inflammatory drug, in 4- to <7-year childrenSteroids · 2025
8. Biallelic variants in ARHGAP19 cause a progressive inherited motor-predominant neuropathyJournal of Clinical Investigation · 2025
9. Del-Zota Produced Statistically Significant Increases in Exon Skipping and Dystrophin Levels in EXPLORE44: A Phase 1/2 Study in Patients with DMD44Neuropediatrics · 2025
10. Long‐Term Evaluation of Givinostat in Duchenne Muscular Dystrophy, and Natural History ComparisonsAnnals of Clinical and Translational Neurology · 2025
11. The parasagittal dural space of the human brainBrain · 2025
12. Pamrevlumab did not meet its primary endpoint for ambulatory patients with Duchenne Muscular Dystrophy: the LELANTOS-2 trial2025
13. Pamrevlumab did not meet its primary endpoint for non-ambulatory patients with Duchenne Muscular Dystrophy: the LELANTOS-1 trial2025
14. Cost comparison of F(ab’) 2 and Fab antivenoms for pit viper envenomation in the United States: a real-world analysisExpert Review of Pharmacoeconomics & Outcomes Research · 2025
15. Gold Mesoporous Silica-Coated Nanoparticles for Quantifying and Qualifying Mesenchymal Stem Cell Distribution; a Proof-of-Concept Study in Large AnimalsACS Applied Bio Materials · 2025
16. Genotype-specific effects of elamipretide in patients with primary mitochondrial myopathy: a post hoc analysis of the MMPOWER-3 trialOrphanet Journal of Rare Diseases · 2024
17. Results of the ACTION‐Galactosemia Kids Study to Evaluate the Effects of Govorestat in Pediatric Patients with Classic GalactosemiaThe Journal of Clinical Pharmacology · 2024
18. Effects of EDG-5506, a Fast Myosin Modulator, on Function and Biomarkers of Muscle Damage in Adults with Becker Muscular Dystrophy (S21.002)Neurology · 2024
19. Comparison of Efficacy between Topical Luliconazole and Topical Clotrimazole in the Treatment of Tinea Corporis and Tinea CrurisThe Journal of Sylhet Women’s Medical College · 2023
20. Pamrevlumab, a Fully Human Monoclonal Antibody Targeting Connective Tissue Growth Factor, for Non-Ambulatory Patients with Duchenne Muscular DystrophyJournal of Neuromuscular Diseases · 2023