Exploring concurrent validity of the CLN2 Clinical Rating Scale: Comparison to PedsQL using cerliponase alfa clinical trial data von Specchio, Nicola, Gissen, Paul, de Los Reyes, Emily, Olaye, Andrew, Camp, Charlotte, Curteis, Tristan, Griffiths, Annabel, Butt, Thomas, Cohen-Pfeffer, Jessica, Slasor, Peter, Sisic, Zlatko, Jain, Mohit, Schulz, Angela

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The humanistic burden of hereditary angioedema: Results from the Burden of Illness Study in Europe von Caballero, Teresa, Aygören-Pürsün, Emel, Bygum, Anette, Beusterien, Kathleen, Hautamaki, Emily, Sisic, Zlatko, Wait, Suzanne, Boysen, Henrik B.

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Socioeconomic burden of hereditary angioedema: results from the hereditary angioedema burden of illness study in Europe von Aygören-Pürsün, Emel, Bygum, Anette, Beusterien, Kathleen, Hautamaki, Emily, Sisic, Zlatko, Wait, Suzanne, Boysen, Henrik B, Caballero, Teresa

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Investigating health-related quality of life in rare diseases: a case study in utility value determination for patients with CLN2 disease (neuronal ceroid lipofuscinosis type 2) von Gissen, Paul, Specchio, Nicola, Olaye, Andrew, Jain, Mohit, Butt, Thomas, Ghosh, Wrik, Ruban-Fell, Benjamin, Griffiths, Annabel, Camp, Charlotte, Sisic, Zlatko, Schwering, Christoph, Wibbeler, Eva, Trivisano, Marina, Lee, Laura, Nickel, Miriam, Mortensen, Amanda, Schulz, Angela

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Acetylator Status Impacts Amifampridine Phosphate (Firdapse™) Pharmacokinetics and Exposure to a Greater Extent Than Renal Function von Haroldsen, Peter E., PhD, Sisic, Zlatko, MD, Datt, Joe, MD, Musson, Donald G., PhD, Ingenito, Gary, MD, PhD

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Pregnancy in patients with mucopolysaccharidosis: a case series von Stewart, Fiona J., Bentley, Andrew, Burton, Barbara K., Guffon, Nathalie, Hale, Susan L., Harmatz, Paul R., Kircher, Susanne G., Kochhar, Pavan K., Mitchell, John J., Plöckinger, Ursula, Graham, Sue, Sande, Stephen, Sisic, Zlatko, Johnston, Tracey A.

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Burden of Illness in Hereditary Angioedema: A Conceptual Model von Bygum, Anette, Aygören-Pürsün, Emel, Beusterien, Kathleen, Hautamaki, Emily, Sisic, Zlatko, Wait, Suzanne, Boysen, Henrik B, Caballero, Teresa

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Expert Opinions on Managing Fertility and Pregnancy in Patients With Mucopolysaccharidosis von Stewart, Fiona, Bentley, Andrew, Burton, Barbara K, Guffon, Nathalie, Hale, Susan L., Harmatz, Paul R., Kircher, Susanne G., Kochhar, Pavan K., Mitchell, John J., Plöckinger, Ursula, Semotok, Jennifer, Graham, Sue, Sande, Stephen, Sisic, Zlatko, Johnston, Tracey A.

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Enzyme replacement therapy initiated in adulthood: Findings from the mucopolysaccharidosis VI Clinical Surveillance Program von Lampe, Christina, Harmatz, Paul R., Parini, Rossella, Sharma, Reena, Teles, Elisa Leão, Johnson, Julie, Sivam, Debbie, Sisic, Zlatko

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Enzyme replacement therapy outcomes across the disease spectrum: Findings from the mucopolysaccharidosis VI Clinical Surveillance Program von Harmatz, Paul R., Lampe, Christina, Parini, Rossella, Sharma, Reena, Teles, Elisa L., Johnson, Julie, Sivam, Debbie, Sisic, Zlatko

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Enzyme replacement therapy in patients with mucopolysaccharidosis type VI: Updated findings from the MPS VI clinical surveillance program von Harmatz, Paul, Lampe, Christina, Parini, Rossella, Sharma, Reena, Leão-Teles, Elisa, Johnson, Julie, Sivam, Debbie, Sisic, Zlatko

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Mucopolysaccharidosis type VI enzyme replacement therapy initiated in adulthood: Findings from the MPS VI clinical surveillance program von Harmatz, Paul, Lampe, Christina, Parini, Rossella, Sharma, Reena, Teles, Elisa Leão, Hawley, Sara, Johnson, Julie, Sivam, Debbie, Sisic, Zlatko

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Mucopolysaccharidosis type VI enzyme replacement therapy outcomes across the disease spectrum: findings from the MPS VI clinical surveillance program von Harmatz, Paul, Lampe, Christina, Parini, Rossella, Sharma, Reena, Teles, Elisa Leão, Hawley, Sara, Johnson, Julie, Sivam, Debbie, Sisic, Zlatko

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Cervical cord compression in mucopolysaccharidosis VI (MPS VI): Findings from the MPS VI Clinical Surveillance Program (CSP) von Solanki, Guirish A., Sun, Peter P., Martin, Kenneth W., Hendriksz, Christian J., Lampe, Christina, Guffon, Nathalie, Hung, Annie, Sisic, Zlatko, Shediac, Renée, Harmatz, Paul R.

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Time Trade-Off Utilities for Hereditary Angioedema Health and Caregiver States von Lo, Siu Hing, Lloyd, Andrew, Elkhalifa, Shuayb, Sisic, Zlatko, van Nooten, Floortje E.

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Impact of elosulfase alfa in patients with morquio A syndrome who have limited ambulation: An open‐label, phase 2 study von Harmatz, Paul R., Mengel, Eugen, Geberhiwot, Tarekegn, Muschol, Nicole, Hendriksz, Christian J., Burton, Barbara K., Jameson, Elisabeth, Berger, Kenneth I., Jester, Andrea, Treadwell, Marsha, Sisic, Zlatko, Decker, Celeste

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Estimation of EuroQol 5-Dimensions health status utility values in hereditary angioedema von Aygören-Pürsün, Emel, Bygum, Anette, Beusterien, Kathleen, Hautamaki, Emily, Sisic, Zlatko, Boysen, Henrik B, Caballero, Teresa

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Burden of Illness in Hereditary Angioedema: A Conceptual Model von Bygum, A, Aygören-Pürsün, E, Beusterien, K, Hautamaki, E, Sisic, Z, Wait, S, Boysen, H, Caballero, T

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Impact of elosulfase alfa in patients with Morquio syndrome type A who have limited ambulation: An open-label, phase 2 study von Harmatz, Paul R., Jester, Andrea, Mengel, Eugen, Treadwell, Marsha, Burton, Barbara K., Berger, Kenneth I., Hendriksz, Christian J., Geberhiwot, Tarekegn, Sisic, Zlatko, Decker, Celeste

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Management of fertility and pregnancy in individuals with mucopolysaccharidosis (MPS) von Stewart, Fiona, Harmatz, Paul, Braunlin, Elizabeth, Bentley, Andrew, Burton, Barbara, Guffon, Nathalie, Hale, Susan, Johnston, Tracey, Kircher, Susanne, Kochhar, Pavan, Mitchell, John, Plöckinger, Ursula, Semotok, Jennifer, Sisic, Zlatko

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