Psychosocial impact on siblings of patients with developmental and epileptic encephalopathies von Bailey, Laurie D., Schwartz, Lauren, Dixon-Salazar, Tracy, Meskis, Mary Anne, Galer, Bradley S., Gammaitoni, Arnold R., Schad, Carla

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Acute Progression of Neuromuscular Findings in Infantile Pompe Disease von Burrow, T. Andrew, MD, Bailey, Laurie A., MS, Kinnett, Douglas G., MD, Hopkin, Robert J., MD

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Comparison of health-related quality of life between heterozygous women with Fabry disease, a healthy control population, and patients with other chronic disease von Street, Natalie Jansen, Yi, Michael S, Bailey, Laurie A, Hopkin, Robert J

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Arrhythmia and Clinical Cardiac Findings in Children With Anderson-Fabry Disease von Wilson, Hunter C., Hopkin, Robert J., Madueme, Peace C., Czosek, Richard J., Bailey, Laurie A., Taylor, Michael D., Jefferies, John L.

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Validation of the PedsQL Epilepsy Module: A pediatric epilepsy‐specific health‐related quality of life measure von Modi, Avani C., Junger, Katherine F., Mara, Constance A., Kellermann, Tanja, Barrett, Lauren, Wagner, Janelle, Mucci, Grace A., Bailey, Laurie, Almane, Dace, Guilfoyle, Shanna M., Urso, Lauryn, Hater, Brooke, Hustzi, Heather, Smith, Gigi, Herrmann, Bruce, Perry, M. Scott, Zupanc, Mary, Varni, James W.

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Novel neurological findings in an adult patient with Gaucher disease von Hagen, Leanne, Bailey, Laurie, Kisanuki, Yaz Y., Prada, Carlos

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Evaluation of drug-endangered children: The yield of toxicology and skeletal survey screening von Howell, Sabrie, Bailey, Laurie, Coffman, Jamye

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The importance of the autopsy in understanding rare disease: Defining the role of the genetics team von Bailey, Laurie, Serbinski, Carolyn R.

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64 Quality of life for pediatric patients with Fabry disease von Hopkin, Robert, Taylor, Heather M., Bailey, Laurie A., Yi, Michael S.

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PEDiDBS: The Pediatric International Deep Brain Stimulation Registry Project von Marks, Warren, Bailey, Laurie

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Pain and fatigue associated with generalized joint hypermobility in Gaucher disease von Mahan, Farrah R., Bailey, Laurie, Pilipenko, Valentina, Prada, Carlos

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Utility of multiple myeloma screening in Gaucher disease von Prada, Carlos, Abell, Katherine, Chadwell, Sarah, Bailey, Laurie

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Safety of enzyme replacement therapy to treat Hunter syndrome in children aged 5 years and under von Grant, Nathan, Bailey, Laurie, Prada, Carlos

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Female sexual dysfunction: A nontraditional risk factor for cardiovascular disease von Bailey, Laurie A., Hoffman, Donna

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Could it be something else? Patients with Gaucher disease, chronic pain, and fatigue found to also meet diagnostic criteria for another inherited condition, Ehlers-Danlos syndrome,... von Bailey, Laurie, Andrew Burrow, T., Wehmeyer, Connie

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Support for siblings of children with lysosomal disorders von Berry, Lisa, Bailey, Laurie, Wehmeyer, Connie

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Long term outcome of bone marrow transplantation for Hunter syndrome: A case report von Berry, Lisa, Bailey, Laurie, Andrew Burrow, T., Wehmeyer, Connie

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A single center’s experience with hematopoietic stem cell transplant in MPS IH from 2002-2015: A retrospective review of variables that may influence transplant outcome and complic... von Wehmeyer, Connie, Bailey, Laurie, Berry, Lisa, Leslie, Nancy

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Effect of enzyme replacement therapy on airway abnormalities in patients with Hunter syndrome von Wood, Robert E., Hopkin, Robert, Wehmeyer, Connie, Bailey, Laurie

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Combination therapy (eliglustat + velaglucerase alfa) in a pediatric patient with Gaucher disease type 1 and hereditary spherocytosis von Bailey, Laurie, Ambruso, Daniel, Grabowski, Gregory, Burrow, Thomas A.

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