2020
OR29-01 Long-Term Safety in Adults with X-Linked Hypophosphatemia (XLH) Treated with Burosumab, a Fully Human Monoclonal Antibody Against FGF23: Final Results of a Phase 3 Trial
Perwad F, Portale A, Carpenter T, Briot K, Imel E, Kamenicky P, Weber T, Pitukcheewanont P, Cheong H, De Beur S, Imanishi Y, Ito N, Lachmann R, Tanaka H, Zhang L, Skrinar A, Rees L, Insogna K. OR29-01 Long-Term Safety in Adults with X-Linked Hypophosphatemia (XLH) Treated with Burosumab, a Fully Human Monoclonal Antibody Against FGF23: Final Results of a Phase 3 Trial. Journal Of The Endocrine Society 2020, 4: or29-01. PMCID: PMC7209551, DOI: 10.1210/jendso/bvaa046.147.Peer-Reviewed Original ResearchYears of ageSerum phosphorusWeek 96Dose reductionLong-term safety resultsTreatment-emergent adverse eventsMonoclonal antibodiesBaseline serum phosphorusHuman IgG1 monoclonal antibodyMean iPTH levelPhase 3 trialLong-term safetyHuman monoclonal antibodyConditional marketing authorizationMonths of ageIgG1 monoclonal antibodyIPTH levelsPlacebo groupAdverse eventsTreatment withdrawalWeek 24Radiographic evidenceMulticenter studyBurosumab treatmentMild hyperphosphatemia
2004
Multisystem study of 20 older adults with Williams syndrome
Cherniske EM, Carpenter TO, Klaiman C, Young E, Bregman J, Insogna K, Schultz RT, Pober BR. Multisystem study of 20 older adults with Williams syndrome. American Journal Of Medical Genetics Part A 2004, 131A: 255-264. PMID: 15534874, DOI: 10.1002/ajmg.a.30400.Peer-Reviewed Original ResearchConceptsStandard oral glucose tolerance testingOral glucose tolerance testingNatural historyLong-term natural historyHigh-frequency sensorineural hearing lossFrequency sensorineural hearing lossOlder adultsAbnormal body habitusWilliams syndromeAbnormal glucose toleranceGlucose tolerance testingBone mineral densitySensorineural hearing lossMultiple organ systemsBrain MRI scansYears of ageDiverticular diseaseGastrointestinal symptomsSubclinical hypothyroidismGlucose toleranceMultimodal therapyDEXA scanningBody habitusMineral densityCardiovascular disease