Physician
Pediatrics
Dr. Emil D. Kakkis, MD
Emil Kakkis works in the field of Pediatrics. Emil Kakkis is also a published doctor. He has 43 paper published. The most recent paper is: Retrovirus-mediated transfer of the human alpha-L-iduronidase cDNA into human hematopoietic progenitor cells leads to correction in trans of Hurler fibroblasts.
Publications
- First human treatment with investigational rhGUS enzyme replacement therapy in an advanced stage MPS VII patient.
- Mannose 6-phosphate conjugation is not sufficient to allow induction of immune tolerance to phenylalanine ammonia-lyase in dogs.
- The Potential Investment Impact of Improved Access to Accelerated Approval on the Development of Treatments for Low Prevalence Rare Diseases.
- Arterial pathology in canine mucopolysaccharidosis-I and response to therapy.
- A dose-optimization trial of laronidase (Aldurazyme) in patients with mucopolysaccharidosis I.
- Long-term efficacy and safety of laronidase in the treatment of mucopolysaccharidosis I.
- Immune tolerance improves the efficacy of enzyme replacement therapy in canine mucopolysaccharidosis I.
- Enzyme replacement therapy in patients who have mucopolysaccharidosis I and are younger than 5 years: results of a multinational study of recombinant human alpha-L-idu...
- A follow-up study of MPS I patients treated with laronidase enzyme replacement therapy for 6 years.
- Hemodynamic changes after protamine administration: association with mortality after coronary artery bypass surgery.
- Enzyme replacement therapy for mucopolysaccharidosis I: a randomized, double-blinded, placebo-controlled, multinational study of recombinant human alpha-L-iduronidase ...
- Efficacy of sapropterin dihydrochloride in increasing phenylalanine tolerance in children with phenylketonuria: a phase III, randomized, double-blind, placebo-controll...
- Penetration, diffusion, and uptake of recombinant human alpha-L-iduronidase after intraventricular injection into the rat brain.
- Identification and molecular characterization of alpha-L-iduronidase mutations present in mucopolysaccharidosis type I patients undergoing enzyme replacement therapy.
- Immune tolerance after long-term enzyme-replacement therapy among patients who have mucopolysaccharidosis I.
- Enzyme replacement therapy for the mucopolysaccharide storage disorders.
- Enzyme replacement therapy in feline mucopolysaccharidosis I.
- Enzyme-replacement therapy in mucopolysaccharidosis I.
- Carbohydrate structures of recombinant human alpha-L-iduronidase secreted by Chinese hamster ovary cells.
- Long-term and high-dose trials of enzyme replacement therapy in the canine model of mucopolysaccharidosis I.
- DOOR syndrome (deafness, onycho-osteodystrophy, and mental retardation): a new patient and delineation of neurologic variability among recessive cases.
- Acute lymphocytic leukemia in acrodermatitis enteropathica.
- Overexpression of the human lysosomal enzyme alpha-L-iduronidase in Chinese hamster ovary cells.
- Enzyme replacement in a canine model of Hurler syndrome.
- A plasmacytoma-specific factor binds the c-myc promoter region.
- A repressor of c-myc transcription is found specifically in plasmacytomas.
- Transcription factors in terminally differentiated B cells.
- An active chromatin structure acquired by translocated c-myc genes.
- Strong transcriptional activation of translocated c-myc genes occurs without a strong nearby enhancer or promoter.
- A transcriptional repressor of c-myc.
- Retrovirus-mediated transfer of the human alpha-L-iduronidase cDNA into human hematopoietic progenitor cells leads to correction in trans of Hurler fibroblasts.
- Intrathecal enzyme replacement therapy reduces lysosomal storage in the brain and meninges of the canine model of MPS I.
- Successful induction of immune tolerance to enzyme replacement therapy in canine mucopolysaccharidosis I.
- Enzyme replacement therapy for mucopolysaccharidosis VI: a phase 3, randomized, double-blind, placebo-controlled, multinational study of recombinant human N-acetylgala...
- Intrathecal administration of AAV vectors for the treatment of lysosomal storage in the brains of MPS I mice.
- Overcoming the blood-brain barrier with high-dose enzyme replacement therapy in murine mucopolysaccharidosis VII.
- Intrathecal enzyme replacement therapy: successful treatment of brain disease via the cerebrospinal fluid.
- Intrathecal enzyme replacement therapy in a patient with mucopolysaccharidosis type I and symptomatic spinal cord compression.
- Research challenges in central nervous system manifestations of inborn errors of metabolism.
- Enzyme replacement in a human model of mucopolysaccharidosis IVA in vitro and its biodistribution in the cartilage of wild type mice.
- Intrathecal administration of recombinant human N-acetylgalactosamine 4-sulfatase to a MPS VI patient with pachymeningitis cervicalis.
- Repeated intrathecal injections of recombinant human 4-sulphatase remove dural storage in mature mucopolysaccharidosis VI cats primed with a short-course tolerisation ...
Schools
University of California At Los Angeles
Doctors Specialties
- Clinical Genetics
- Pediatrics
Accepted Insurances
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