C. Decker

发表

Antibodies that neutralize cellular uptake of elosulfase alfa are not associated with reduced efficacy or pharmacodynamic effect in individuals with Morquio A syndrome.

B. Schweighardt, A. Bagri, C. O’Neill, 2017, Journal of immunological methods.

Impact of long-term elosulfase alfa on activities of daily living in patients with Morquio A syndrome in an open-label, multi-center, phase 3 extension study.

John J. Mitchell, Robert A. Matousek, B. Burton, 2017, Molecular genetics and metabolism.

Induction, titration, and maintenance dosing regimen in a phase 2 study of pegvaliase for control of blood phenylalanine in adults with phenylketonuria.

B. Schweighardt, K. Larimore, N. Longo, 2018, Molecular genetics and metabolism.

Longitudinal analysis of endurance and respiratory function from a natural history study of Morquio A syndrome.

John J. Mitchell, P. Slasor, B. Burton, 2015, Molecular genetics and metabolism.

Galsulfase (Naglazyme®) therapy in infants with mucopolysaccharidosis

L. Randolph, P. Harmatz, N. Guffon, 2014 .

Galsulfase (Naglazyme®) therapy in infants with mucopolysaccharidosis VI

L. Randolph, P. Garcia, P. Harmatz, 2013, Journal of Inherited Metabolic Disease.

Safety, immunogenicity, and clinical outcomes in patients with Morquio A syndrome participating in 2 sequential open-label studies of elosulfase alfa enzyme replacement therapy (MOR-002/MOR-100), representing 5 years of treatment.

C. Hendriksz, S. Santra, Yulan Qi, 2018, Molecular genetics and metabolism.

Long-term Immunogenicity of Elosulfase Alfa in the Treatment of Morquio A Syndrome: Results From MOR-005, a Phase III Extension Study.

B. Schweighardt, P. Slasor, C. O’Neill, 2017, Clinical therapeutics.

Pegvaliase for the treatment of phenylketonuria: Results of the phase 2 dose-finding studies with long-term follow-up.

K. Larimore, J. Vockley, B. Burton, 2020, Molecular genetics and metabolism.

Enzyme replacement therapy for mucopolysaccharidosis VI: long-term cardiac effects of galsulfase (Naglazyme®) therapy

G. Gildengorin, R. Giugliani, J. E. Wraith, 2012, Journal of Inherited Metabolic Disease.

Long-term follow-up of endurance and safety outcomes during enzyme replacement therapy for mucopolysaccharidosis VI: Final results of three clinical studies of recombinant human N-acetylgalactosamine 4-sulfatase.

P. Kaplan, R. Steiner, R. Giugliani, 2008, Molecular genetics and metabolism.

Biomarker analysis of Morquio syndrome: identification of disease state and drug responsive markers

Kelly Lau, C. Decker, E. Foehr, 2011, Orphanet journal of rare diseases.

Successful Management of Difficult Infusion-Associated Reactions in a Young Patient With Mucopolysaccharidosis Type VI Receiving Recombinant Human Arylsulfatase B (Galsulfase [Naglazyme])

B. Burton, C. Decker, Katherine H. Kim, 2008, Pediatrics.

The Morquio A Clinical Assessment Program: baseline results illustrating progressive, multisystemic clinical impairments in Morquio A subjects.

John J. Mitchell, P. Slasor, B. Burton, 2013, Molecular genetics and metabolism.

Impact of long-term elosulfase alfa treatment on respiratory function in patients with Morquio A syndrome

John J. Mitchell, Robert A. Matousek, B. Burton, 2016, Journal of Inherited Metabolic Disease.

Enzyme replacement therapy for mucopolysaccharidosis VI: evaluation of long-term pulmonary function in patients treated with recombinant human N-acetylgalactosamine 4-sulfatase

Zi-Fan Yu, Maurizio Scarpa, Paul Harmatz, 2010, Journal of Inherited Metabolic Disease.

Enzyme replacement therapy for mucopolysaccharidosis VI: Growth and pubertal development in patients treated with recombinant human N-acetylgalactosamine 4-sulfatase.

P. Kaplan, R. Steiner, R. Giugliani, 2010, Journal of pediatric rehabilitation medicine.

Long-term safety and efficacy of pegvaliase for the treatment of phenylketonuria in adults: combined phase 2 outcomes through PAL-003 extension study

K. Larimore, J. Vockley, B. Burton, 2018, Orphanet Journal of Rare Diseases.

Relationship Between Patient-Reported Outcomes and Clinical Outcomes in Patients With Morquio A Syndrome

Christina Lampe, Eugen Mengel, A. Olaye, 2015 .

Impact of elosulfase alfa in patients with morquio A syndrome who have limited ambulation: An open‐label, phase 2 study

B. Burton, C. Hendriksz, E. Jameson, 2016, American journal of medical genetics. Part A.

BMN 110 (Recombinant Human GALNS), an Investigational Enzyme Replacement Therapy for Mucopolysaccharidosis Type IVA (MPS IVA Or Morquio Syndrome), Promotes Type II Collagen Synthesis in MPS IVA Patients

C. Hendriksz, C. O’Neill, L. Tsuruda, 2012 .

Long Term Outcomes of a Phase 1/2, Multicenter, Open-Label, Dose-Escalation Study to Evaluate the Safety, Tolerability, and Efficacy of BMN 110 in Patients with Mucopolysaccharidosis IVA (Morquio A Syndrome)

C. Hendriksz, A. Vellodi, C. Decker, 2012 .

Longitudinal analysis of endurance and respiratory function from a natural history study of Morquio A syndrome.

John J. Mitchell, P. Slasor, B. Burton, 2015, Molecular genetics and metabolism.