A multicenter, open-label study evaluating safety and clinical outcomes in children (1.4-7.5 years) with Hunter syndrome receiving idursulfase enzyme replacement therapy
Giugliani, Roberto, Hwu, Wuh-Liang, Tylki-Szymanska, Anna, Whiteman, David A H, Pano, Arian
Published in Genetics in medicine (01.06.2014)
Published in Genetics in medicine (01.06.2014)
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Effects of sulfatide on peripheral nerves in metachromatic leukodystrophy
Farah, Mohamed H., Dali, Christine í, Groeschel, Samuel, Moldovan, Mihai, Whiteman, David A. H., Malanga, C. J., Krägeloh‐Mann, Ingeborg, Li, Jing, Barton, Norman, Krarup, Christian
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Published in Annals of clinical and translational neurology (01.02.2024)
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Diagnosis and management of defects of mitochondrial beta-oxidation
Vockley, Jerry, Singh, Rani H, Whiteman, David A H
Published in Current opinion in clinical nutrition and metabolic care (01.11.2002)
Published in Current opinion in clinical nutrition and metabolic care (01.11.2002)
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Clinical investigator perspectives on the effects of intrathecal idursulfase-IT treatment in children with neuronopathic mucopolysaccharidosis type II
Yee, Karen S., Alexanderian, David, Hamm, Lynne, Martin, Susan, Olayinka-Amao, Bimpe, Whiteman, David A.H.
Published in Molecular genetics and metabolism (01.02.2022)
Published in Molecular genetics and metabolism (01.02.2022)
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Novel insights into mucopolysaccharidosis type II based on an analysis of genetic variants in 763 patients
Ayodele, Olulade, Beetz, Christian, Bauer, Peter, Yee, Karen S., Fertek, Daniel, Audi, Jennifer, Whiteman, David A.H.
Published in Molecular genetics and metabolism (01.02.2022)
Published in Molecular genetics and metabolism (01.02.2022)
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Genotype–phenotype findings in patients with mucopolysaccharidosis II from the Hunter Outcome Survey
Muenzer, Joseph, Amartino, Hernan, Burton, Barbara K., Scarpa, Maurizio, Tylki-Szymańska, Anna, Audi, Jennifer, Botha, Jaco, Fertek, Daniel, Merberg, David, Natarajan, Madhusudan, Whiteman, David A.H., Giugliani, Roberto
Published in Molecular genetics and metabolism (01.09.2024)
Published in Molecular genetics and metabolism (01.09.2024)
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Cognitive and adaptive behaviors associated with disease severity and genotype in patients with mucopolysaccharidosis II
Yee, Karen S., Alexanderian, David, Merberg, David, Natarajan, Madhusudan, Wang, Scarlett, Wu, Yuna, Whiteman, David A.H.
Published in Molecular genetics and metabolism (01.11.2023)
Published in Molecular genetics and metabolism (01.11.2023)
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Intrathecal idursulfase-IT in patients with neuronopathic mucopolysaccharidosis II: Results from a phase 2/3 randomized study
Muenzer, Joseph, Burton, Barbara K., Harmatz, Paul, Gutiérrez-Solana, Luis González, Ruiz-Garcia, Matilde, Jones, Simon A., Guffon, Nathalie, Inbar-Feigenberg, Michal, Bratkovic, Drago, Hale, Michael, Wu, Yuna, Yee, Karen S., Whiteman, David A.H., Alexanderian, David
Published in Molecular genetics and metabolism (01.09.2022)
Published in Molecular genetics and metabolism (01.09.2022)
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A phase 2 study assessing TAK-611150 mg intrathecal weekly in patients with late-infantile metachromatic leukodystrophy (SHP611–201; EMBOLDEN) compared to matched historical control data from children with late-infantile MLD (GLIA-MLD)
Adang, Laura A., Malanga, C.J., Burton, Barbara K., Giugliani, Roberto, Groeschel, Samuel, Inbar-Feigenberg, Michal, Sevin, Caroline, Riera, Mireia Del Toro, Yin, Shaoming, Rathmann, Suchitrita, Whiteman, David A.H., Vanderver, Adeline
Published in Molecular genetics and metabolism (01.02.2024)
Published in Molecular genetics and metabolism (01.02.2024)
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The effect of intrathecal recombinant arylsulfatase A therapy on demyelination load in children with metachromatic leukodystrophy
Groeschel, Samuel, Beerepoot, Shanice, Amedick, Lucas Bastian, Krӓgeloh-Mann, Ingeborg, Li, Jing, Whiteman, David A.H., Wolf, Nicole I., Port, John D.
Published in Molecular genetics and metabolism (01.02.2022)
Published in Molecular genetics and metabolism (01.02.2022)
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The effect of intrathecal recombinant arylsulfatase A therapy on structural brain magnetic resonance imaging in children with metachromatic leukodystrophy
Groeschel, Samuel, Beerepoot, Shanice, Amedick, Lucas Bastian, Krӓgeloh‐Mann, Ingeborg, Li, Jing, Whiteman, David A. H., Wolf, Nicole I., Port, John D.
Published in Journal of inherited metabolic disease (01.07.2024)
Published in Journal of inherited metabolic disease (01.07.2024)
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Long-term open-label extension study of the safety and efficacy of intrathecal idursulfase-IT in patients with neuronopathic mucopolysaccharidosis II
Muenzer, Joseph, Burton, Barbara K., Harmatz, Paul, Gutiérrez-Solana, Luis González, Ruiz-Garcia, Matilde, Jones, Simon A., Guffon, Nathalie, Inbar-Feigenberg, Michal, Bratkovic, Drago, Hale, Michael, Wu, Yuna, Yee, Karen S., Whiteman, David A.H., Alexanderian, David
Published in Molecular genetics and metabolism (01.09.2022)
Published in Molecular genetics and metabolism (01.09.2022)
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Corrigendum to: Intrathecal idursulfase-IT in patients with neuronopathic mucopolysaccharidosis II: Results from a phase 2/3 randomized study
Muenzer, Joseph, Burton, Barbara K., Harmatz, Paul, Gutiérrez-Solana, Luis González, Ruiz-Garcia, Matilde, Jones, Simon A., Guffon, Nathalie, Inbar-Feigenberg, Michal, Bratkovic, Drago, Hale, Michael, Wu, Yuna, Yee, Karen S., Whiteman, David A.H., Alexanderian, David
Published in Molecular genetics and metabolism (01.11.2023)
Published in Molecular genetics and metabolism (01.11.2023)
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Corrigendum to “Long-term open-label extension study of the safety and efficacy of intrathecal idursulfase-IT in patients with neuronopathic mucopolysaccharidosis II”
Muenzer, Joseph, Burton, Barbara K., Harmatz, Paul, Gutiérrez-Solana, Luis González, Ruiz-Garcia, Matilde, Jones, Simon A., Guffon, Nathalie, Inbar-Feigenberg, Michal, Bratkovic, Drago, Hale, Michael, Wu, Yuna, Yee, Karen S., Whiteman, David A.H., Alexanderian, David
Published in Molecular genetics and metabolism (01.11.2023)
Published in Molecular genetics and metabolism (01.11.2023)
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Clinical investigator perspectives on patient outcomes in children with neuronopathic mucopolysaccharidosis II during intrathecal idursulfase-IT treatment
Yee, Karen S, Alexanderian, David, Martin, Susan, Olayinka-Amao, Bimpe, Whiteman, David A H
Published in Orphanet journal of rare diseases (12.04.2024)
Published in Orphanet journal of rare diseases (12.04.2024)
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Fifteen years of the Hunter Outcome Survey (HOS): Real-world insights into the patient population living with mucopolysaccharidosis type II (MPS II)
Muenzer, Joseph, Scarpa, Maurizio, Tylki-Szymańska, Anna, Amartino, Hernan, Harmatz, Paul, Lin, Shuan-Pei, Link, Bianca, Molter, David, Raiman, Julian, Whiteman, David A.H., Botha, Jaco, Jazukeviciene, Dalia, Fertek, Daniel, Audi, Jennifer, Burton, Barbara
Published in Molecular genetics and metabolism (01.02.2022)
Published in Molecular genetics and metabolism (01.02.2022)
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Infusion-related reactions in patients with mucopolysaccharidosis type II on idursulfase enrolled in the Hunter Outcome Survey (HOS)
Burton, Barbara K., Lampe, Christina, Lagler, Florian B., Botha, Jaco, Whiteman, David A.H.
Published in Molecular genetics and metabolism (01.02.2018)
Published in Molecular genetics and metabolism (01.02.2018)
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