Unveiling the untreated: development of a database algorithm to identify potential Fabry disease patients in Germany von Hilz, Max J, Lyn, Nicole, Marczykowski, Felix, Werner, Barbara, Pignot, Marc, Ponce, Elvira, Bender, Joseph, Edigkaufer, Michael, DasMahapatra, Pronabesh

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Development of the Fabry Disease Patient-Reported Outcome (FD-PRO): a new instrument to measure the symptoms and impacts of Fabry Disease von Hamed, Alaa, DasMahapatra, Pronabesh, Lyn, Nicole, Gwaltney, Chad, Hopkin, Robert J.

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Patient and caregiver perspectives on burden of disease manifestations in late-onset Tay-Sachs and Sandhoff diseases von Lyn, Nicole, Pulikottil-Jacob, Ruth, Rochmann, Camille, Krupnick, Robert, Gwaltney, Chad, Stephens, Nick, Kissell, Julie, Cox, Gerald F., Fischer, Tanya, Hamed, Alaa

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Fabry Disease Patient-Reported Outcome (FD-PRO) demonstrates robust measurement properties for assessing symptom severity in Fabry disease von Hamed, Alaa, DasMahapatra, Pronabesh, Lyn, Nicole, Gwaltney, Chad, Iaconangelo, Charlie, Serrano, Daniel, Modur, Vijay, Politei, Juan

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Socioeconomic Status and Child Development: A Meta-Analysis von Letourneau, Nicole Lyn, Duffett-Leger, Linda, Levac, Leah, Watson, Barry, Young-Morris, Catherine

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Association Between Nonparenting Adult’s Attachment Patterns and Brain Structure and Function von Letourneau, Nicole Lyn, Martha, Hart J, MacMaster, Frank P

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Assessing the impact of pain on health-related quality of life (HRQoL) in patients with Fabry disease von Lyn, Nicole, Wang, Jennifer, Ba, Sarah, Wing, Synne, Pulikottil-Jacob, Ruth, DasMahapatra, Pronabesh

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Measuring the intangible cost of lysosomal disorders: Fabry disease, Gaucher disease type 3, and GM2 gangliosidosis von Lyn, Nicole, Ba, Sarah, Graham, Christina A., Sowell, France, Ruzich, Emily, Krupnick, Robert, Pulikottil-Jacob, Ruth

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Comparing treatment options for Fabry disease: Feasibility assessment for network meta-analysis (NMA) von Lyn, Nicole, Guyot, Patricia, Thakker, Divyesh, Lee, Chae Sung, Maski, Manish, Crespo, Ana, Pulikottil-Jacob, Ruth

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Cluster analysis of kidney function decline among males with Fabry disease in a large United States electronic health records database von Chiorean, Alexandra, Lyn, Nicole, Kabadi, Shaum, Blanchon, Margot, Hayat, Paul, Loustalot, Paul, Maski, Manish, Montmerle, Martin, Ponce, Elvira

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Symptoms of Fabry disease in adolescents von Warsi, Ibrahim, Lyn, Nicole, Whyte, Julie, Witherspoon, Brooke, White, Kailee, DasMahapatra, Pronabesh

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Clustered analysis of Fabry disease progression in a large US electronic health records database: A retrospective observational cohort study von Chiorean, Alexandra, Kabadi, Shaum, Hayat, Paul, Montmerle, Martin, Ponce, Elvira, Blanchon, Margot, Lyn, Nicole, Maski, Manish

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Neuropathic and abdominal pain items of the Fabry Disease Patient-Reported Outcome (FD-PRO) show robust measurement properties in treatment naïve Fabry patients von Lyn, Nicole, Laconangelo, Charlie, Serrano, Daniel, Gwaltney, Chad, Kupferman, Joseph, DasMahapatra, Pronabesh

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Establishing the content validity of the Fabry Disease-Patient Reported Outcome (FD-PRO) for adolescent patients with Fabry disease von Lyn, Nicole, Warsi, Ibrahim, Whyte, Julie, Witherspoon, Brooke, Blatt, Elliott, DasMahapatra, Pronabesh

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Clinical relevance of globotriaosylceramide accumulation in Fabry disease and the effect of agalsidase beta in affected tissues von Tøndel, Camilla, Thurberg, Beth L., DasMahapatra, Pronabesh, Lyn, Nicole, Maski, Manish, Batista, Julie L., George, Kelly, Patel, Hiren, Hariri, Ali

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Patients' and Caregivers' Preferences for Different Hemophilia a Treatment Attributes von Joshi, Namita, Ng, Xinyi, Botteman, Marc, Li, Nanxin, Shah, Rachel, Jain, Nisha, Lyn, Nicole, Su, Jun

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Respiration pattern variability and related default mode network connectivity are altered in remitted depression von Zamoscik, Vera Eva, Schmidt, Stephanie Nicole Lyn, Gerchen, Martin Fungisai, Samsouris, Christos, Timm, Christina, Kuehner, Christine, Kirsch, Peter

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A study to evaluate the effect of venglustat on neuropathic and abdominal pain in symptomatic adult patients with Fabry disease von Hopkin, Robert, Kupferman, Joseph, Deegan, Patrick B., Minini, Pascal, Goyeau, Harmonie, Maski, Manish, Lyn, Nicole, DasMahapatra, Pronabesh, Germain, Dominique P.

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Patient and caregiver preferences for haemophilia A treatments: A discrete choice experiment von Su, Jun, Li, Nanxin, Joshi, Namita, Ng, Xinyi, Botteman, Marc, Shah, Rachel, Jain, Nisha, Lyn, Nicole, Preblick, Ronald

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Relevance and comprehension of patient-reported outcome measures of mobility, physical function, and speech among patients with late-onset GM2 gangliosidoses von Pulikottil-Jacob, Ruth, Lyn, Nicole, Rochmann, Camille, Krupnick, Robert, Stephens, Nicholas, Gwaltney, Chad, Hamed, Alaa

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