2023
Life-Saving Treatments for Spinal Muscular Atrophy: Global Access and Availability.
Armengol V, Darras B, Abulaban A, Alshehri A, Barisic N, Ben-Omran T, Bernert G, Castiglioni C, Chien Y, Farrar M, Kandawasvika G, Khadilkar S, Mah J, Marini-Bettolo C, Osredkar D, Pfeffer G, Piazzon F, Pitarch Castellano I, Quijano-Roy S, Saito K, Shin J, Vázquez-Costa J, Walter M, Wanigasinghe J, Xiong H, Griggs R, Roy B. Life-Saving Treatments for Spinal Muscular Atrophy: Global Access and Availability. Neurology Clinical Practice 2023, 14: e200224. PMID: 38107546, PMCID: PMC10723640, DOI: 10.1212/cpj.0000000000200224.Peer-Reviewed Original ResearchSurveyed health care providersProvider survey responsesHealth care providersSpinal muscular atrophyEconomic burdenNewborn screeningBarriers to treatmentMitigate such inequalitiesCare providersClinical careQualitative studyTreated patientsOutcome measuresMotor strengthGenetic testingSpinal muscular atrophy type 1Muscular atrophyProvidersSurvey responsesTreatment of spinal muscular atrophyLife-saving treatmentMuscle weaknessProgressive muscle weaknessNormal lifeTreatment costs
2020
Primary caregiver decision‐making in hematopoietic cell transplantation and gene therapy for sickle cell disease
Sinha C, Bakshi N, Ross D, Loewenstein G, Krishnamurti L. Primary caregiver decision‐making in hematopoietic cell transplantation and gene therapy for sickle cell disease. Pediatric Blood & Cancer 2020, 68: e28749-e28749. PMID: 33034129, PMCID: PMC8246626, DOI: 10.1002/pbc.28749.Peer-Reviewed Original ResearchConceptsHematopoietic cell transplantationSickle cell diseasePrimary caregiversSCD complicationsCell transplantationCell diseaseDiminished qualityAutologous hematopoietic progenitor cellsPrimary caregiver reportMajor medical decisionsGene therapyCurative optionSevere complicationsHematopoietic progenitor cellsClinical trialsAcceptable treatmentRecent complicationsComplicationsCaregiver reportsCaregiversProgenitor cellsNormal lifeTransplantationTherapyMedical decisions
2017
Why People Who Inject Drugs Voluntarily Transition Off Methadone in Ukraine
Rozanova J, Marcus R, Taxman FS, Bojko MJ, Madden L, Farnum SO, Mazhnaya A, Dvoriak S, Altice FL. Why People Who Inject Drugs Voluntarily Transition Off Methadone in Ukraine. Qualitative Health Research 2017, 27: 2057-2070. PMID: 28942704, DOI: 10.1177/1049732317732307.Peer-Reviewed Original Research
2002
Association of Bladder Exstrophy With Congenital Pathology of the Hip and Lumbosacral Spine: A Long-Term Follow-Up Study of 13 Patients
Kaar S, Cooperman D, Blakemore L, Thompson G, Petersilge C, Elder J, Heiple K. Association of Bladder Exstrophy With Congenital Pathology of the Hip and Lumbosacral Spine: A Long-Term Follow-Up Study of 13 Patients. Journal Of Pediatric Orthopaedics 2002, 22: 62-66. DOI: 10.1097/00004694-200201000-00014.Peer-Reviewed Original ResearchLumbosacral spineUrologic repairBladder exstrophyBilateral iliac osteotomyPeriodic radiographic evaluationLong-term followAbnormal radiographic findingsMild acetabular dysplasiaIliac osteotomyRadiographic findingsRadiographic evaluationAcetabular dysplasiaPelvic abnormalitiesPelvic reconstructionCongenital pathologyShort statureSkeletal maturityPatientsNormal lifeSpineExstrophyRepairDysplasiaFollowOsteotomy
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