Search Results - "Cahan, Heather" :: K.UTB vyhledávací portál | TBU Library Retrieval Portal

Characterization of Fluid Biomarkers Reveals Lysosome Dysfunction and Neurodegeneration in Neuronopathic MPS II Patients

Characterization of Fluid Biomarkers Reveals Lysosome Dysfunction and Neurodegeneration in Neuronopathic MPS II Patients

by Bhalla, Akhil, Ravi, Ritesh, Fang, Meng, Arguello, Annie, Davis, Sonnet S, Chiu, Chi-Lu, Blumenfeld, Jessica R, Nguyen, Hoang N, Earr, Timothy K, Wang, Junhua, Astarita, Giuseppe, Zhu, Yuda, Fiore, Damian, Scearce-Levie, Kimberly, Diaz, Dolores, Cahan, Heather, Troyer, Matthew D, Harris, Jeffrey M, Escolar, Maria L
Published in International journal of molecular sciences (22.07.2020)

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Journal Article

Outcomes of Neonatal Candidiasis: The Impact of Delayed Initiation of Antifungal Therapy

Outcomes of Neonatal Candidiasis: The Impact of Delayed Initiation of Antifungal Therapy

by Cahan, Heather, Deville, Jaime G.
Published in International Journal of Pediatrics (01.01.2011)

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Journal Article

Study of Intraventricular Cerliponase Alfa for CLN2 Disease

Study of Intraventricular Cerliponase Alfa for CLN2 Disease

by Schulz, Angela, Ajayi, Temitayo, Specchio, Nicola, de Los Reyes, Emily, Gissen, Paul, Ballon, Douglas, Dyke, Jonathan P, Cahan, Heather, Slasor, Peter, Jacoby, David, Kohlschütter, Alfried
Published in The New England journal of medicine (17.05.2018)

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Journal Article

Immunogenicity to cerliponase alfa intracerebroventricular enzyme replacement therapy for CLN2 disease: Results from a Phase 1/2 study

Immunogenicity to cerliponase alfa intracerebroventricular enzyme replacement therapy for CLN2 disease: Results from a Phase 1/2 study

by Cherukuri, Anu, Cahan, Heather, de Hart, Greg, Van Tuyl, Andrea, Slasor, Peter, Bray, Laurie, Henshaw, Joshua, Ajayi, Temitayo, Jacoby, Dave, O'Neill, Charles A., Schweighardt, Becky
Published in Clinical immunology (Orlando, Fla.) (01.12.2018)

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Journal Article

Cerliponase alfa for the treatment of CLN2 disease in an expanded patient cohort including children younger than three years: Interim results from an ongoing clinical study

Cerliponase alfa for the treatment of CLN2 disease in an expanded patient cohort including children younger than three years: Interim results from an ongoing clinical study

by Schulz, Angela, de los Reyes, Emily, Specchio, Nicola, Gissen, Paul, Cahan, Heather, Slasor, Peter, Ajayi, Temitayo
Published in Molecular genetics and metabolism (01.02.2020)

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Journal Article

Persistent treatment effect of cerliponase alfa in children with CLN2 disease: A 3 year update from an ongoing multicenter extension study

Persistent treatment effect of cerliponase alfa in children with CLN2 disease: A 3 year update from an ongoing multicenter extension study

by Schulz, Angela, Specchio, Nicola, Gissen, Paul, de los Reyes, Emily, Cahan, Heather, Slasor, Peter, Jacoby, David, Ajayi, Temitayo
Published in Molecular genetics and metabolism (01.02.2019)

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Journal Article

Design and preliminary results of a first-in-human, 24-week study of intravenous DNL310 (brain-penetrant IDS) in MPS II

Design and preliminary results of a first-in-human, 24-week study of intravenous DNL310 (brain-penetrant IDS) in MPS II

by Harmatz, Paul, Bakardjiev, Anna I., Escolar, Maria, Burton, Barbara, Muenzer, Joseph, Cahan, Heather, Jones, Simon A., Ryali, Priya, Bhalla, Akhil, Damo, Lorna, Daryani, Vinay M., Harris, Jeffrey M., Zhu, Yuda, Ho, Carole, Troyer, Matthew D.
Published in Molecular genetics and metabolism (01.02.2021)

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Journal Article

Long-term safety and efficacy of intracerebroventricular enzyme replacement therapy with cerliponase alfa in children with CLN2 disease: Two year results from an ongoing multicenter extension study

Long-term safety and efficacy of intracerebroventricular enzyme replacement therapy with cerliponase alfa in children with CLN2 disease: Two year results from an ongoing multicenter extension study

by Schulz, Angela, Specchio, Nicola, Gissen, Paul, de los Reyes, Emily, Cahan, Heather, Slasor, Peter, Ajayi, Temitayo, Jacoby, David
Published in Molecular genetics and metabolism (01.02.2018)

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Journal Article

Evaluation of fluid biomarkers reveals lysosome dysfunction and neurodegeneration in neuronopathic MPS II patients

Evaluation of fluid biomarkers reveals lysosome dysfunction and neurodegeneration in neuronopathic MPS II patients

by Bhalla, Akhil, Ravi, Ritesh, Fang, Meng, Arguello, Annie, Davis, Sonnet S., Chiu, Chi-Lu, Blumenfeld, Jessica R., Nguyen, Hoang N., Earr, Timothy K., Wang, Junhua, Astarita, Giuseppe, Zhu, Yuda, Fiore, Damian, Scearce-Levie, Kimberly, Diaz, Dolores, Cahan, Heather, Troyer, Matthew D., Harris, Jeffrey M., Escolar, Maria L.
Published in Molecular genetics and metabolism (01.02.2021)

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Journal Article

Long-term safety and efficacy of intracerebroventricular enzyme replacement therapy with cerliponase alfa in children with CLN2 disease: interim results from an ongoing multicenter, multinational extension study

Long-term safety and efficacy of intracerebroventricular enzyme replacement therapy with cerliponase alfa in children with CLN2 disease: interim results from an ongoing multicenter, multinational extension study

by Schulz, Angela, Specchio, Nicola, Gissen, Paul, de los Reyes, Emily, Cahan, Heather, Slasor, Peter, Ajayi, Temitayo, Jacoby, David
Published in Molecular genetics and metabolism (01.01.2017)

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Journal Article

ICV-administered BMN 250 (NAGLU-IGF2) is well tolerated and reduces heparan sulfate accumulation in the CNS of subjects with Sanfilippo syndrome type B (MPS IIIB)

ICV-administered BMN 250 (NAGLU-IGF2) is well tolerated and reduces heparan sulfate accumulation in the CNS of subjects with Sanfilippo syndrome type B (MPS IIIB)

by Muschol, Nicole, Cleary, Maureen, Couce, Maria Luz, Shaywitz, Adam J., Cahan, Heather, Grover, Anita, Maricich, Stephen M., Melton, Andrew, Smith, Lynn, de Castro Lopez, Maria Jose
Published in Molecular genetics and metabolism (01.02.2018)

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Journal Article

Immunogenicity to cerliponase alfa, an enzyme replacement therapy for patients with CLN2 disease: results from a phase 1/2 study

Immunogenicity to cerliponase alfa, an enzyme replacement therapy for patients with CLN2 disease: results from a phase 1/2 study

by Cherukuri, Anu, Cahan, Heather, Van Tuyl, Andrea, de Hart, Greg, Slasor, Peter, Bray, Laurie, Henshaw, Josh, Ajayi, Temitayo, Jacoby, Dave, O'Neill, Charles, Schweighardt, Becky
Published in Molecular genetics and metabolism (01.01.2017)

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Journal Article

Natural history data for young subjects with Sanfilippo syndrome type B (MPS IIIB)

Natural history data for young subjects with Sanfilippo syndrome type B (MPS IIIB)

by Okur, Ilyas, Cleary, Maureen, de Castro Lopez, Maria Jose, Harmatz, Paul, Lee, Joy, Lin, Shaun-Pei, Couce, Maria Luz, Muschol, Nicole, Peters, Heidi, Villarreal, Martha Solano, Shaywitz, Adam, Cahan, Heather, Grover, Anita, Maricich, Stephen M., Melton, Andrew, Smith, Lynn, Ezgu, Fatih
Published in Molecular genetics and metabolism (01.02.2018)

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Journal Article

Natural history data for young subjects with Sanfilippo syndrome type B (MPS IIIB)

Natural history data for young subjects with Sanfilippo syndrome type B (MPS IIIB)

by Villarreal, Martha Solano, Okur, Ilyas, Cleary, Maureen, de Castro Lopez, Maria Jose, Harmatz, Paul, Lee, Joy, Lin, Shuan-Pei, Couce, Maria Luz, Muschol, Nicole, Peters, Heidi, Shaywitz, Adam J., Cahan, Heather, Grover, Anita, Melton, Andrew, Smith, Lynn, Maricich, Stephen M., Ezgu, Fatih
Published in Molecular genetics and metabolism (01.02.2019)

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Journal Article

ICV-administered tralesinidase alfa (BMN 250 NAGLU-IGF2) is well-tolerated and reduces heparan sulfate accumulation in the CNS of subjects with Sanfilippo syndrome type B (MPS IIIB)

ICV-administered tralesinidase alfa (BMN 250 NAGLU-IGF2) is well-tolerated and reduces heparan sulfate accumulation in the CNS of subjects with Sanfilippo syndrome type B (MPS IIIB)

by Cleary, Maureen, Muschol, Nicole, Couce, Maria Luz, Harmatz, Paul, Lee, Joy, Lin, Shuan-Pei, Okur, Ilyas, Ezgu, Fatih, Peters, Heidi, Villarreal, Martha Solano, Shaywitz, Adam J., Cahan, Heather, Grover, Anita, Melton, Andrew, Smith, Lynn, Maricich, Stephen M., de Castro Lopez, Maria J.
Published in Molecular genetics and metabolism (01.02.2019)

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Journal Article

METHODS FOR THE TREATMENT OF HUNTER SYNDROME

METHODS FOR THE TREATMENT OF HUNTER SYNDROME

by CAHAN, Heather, GROVER, Anita, TROYER, Matthew D, ARGUELLO, Annie, DARYANI, Vinay M
Year of Publication 29.05.2024

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Patent

METHODS FOR THE TREATMENT OF HUNTER SYNDROME

METHODS FOR THE TREATMENT OF HUNTER SYNDROME

by CAHAN, Heather, GROVER, Anita, TROYER, Matthew D, ARGUELLO, Annie, DARYANI, Vinay M
Year of Publication 23.03.2023

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Patent

METHODS FOR THE TREATMENT OF HUNTER SYNDROME

METHODS FOR THE TREATMENT OF HUNTER SYNDROME

by CAHAN, Heather, GROVER, Anita, TROYER, Matthew D, ARGUELLO, Annie, DARYANI, Vinay M
Year of Publication 14.12.2022

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Patent

METHODS FOR THE TREATMENT OF HUNTER SYNDROME

METHODS FOR THE TREATMENT OF HUNTER SYNDROME

by CAHAN, Heather, GROVER, Anita, TROYER, Matthew D, ARGUELLO, Annie, DARYANI, Vinay M
Year of Publication 28.07.2022

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Patent

METHODS FOR THE TREATMENT OF HUNTER SYNDROME

METHODS FOR THE TREATMENT OF HUNTER SYNDROME

by CAHAN, Heather, GROVER, Anita, TROYER, Matthew D, ARGUELLO, Annie, DARYANI, Vinay M
Year of Publication 12.08.2021

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Patent