| scholarly article | Q13442814 |
| P356 | DOI | 10.1056/NEJMOA032613 |
| P698 | PubMed publication ID | 15128896 |
| P50 | author | Joanne Kurtzberg | Q89893926 |
| Michele D Poe | Q123668390 | ||
| P2093 | author name string | Susan Wood | |
| David A Wenger | |||
| John J Hopwood | |||
| Maria L Escolar | |||
| William Krivit | |||
| Paul Szabolcs | |||
| Young Kim | |||
| Paul L Martin | |||
| Pablo Rubinstein | |||
| Susan L Staba | |||
| June Allison-Thacker | |||
| P433 | issue | 19 | |
| P407 | language of work or name | English | Q1860 |
| P921 | main subject | patient | Q181600 |
| P304 | page(s) | 1960-1969 | |
| P577 | publication date | 2004-05-01 | |
| P1433 | published in | The New England Journal of Medicine | Q582728 |
| P1476 | title | Cord-blood transplants from unrelated donors in patients with Hurler's syndrome | |
| P478 | volume | 350 |
| Q36627142 | A Prospective Natural History Study of Mucopolysaccharidosis Type IIIA. |
| Q45872265 | Abnormal polyamine metabolism is unique to the neuropathic forms of MPS: potential for biomarker development and insight into pathogenesis |
| Q38679561 | Accelerating stem cell trials for Alzheimer's disease |
| Q30479159 | Adult stem cells and their trans-differentiation potential--perspectives and therapeutic applications |
| Q46319967 | Allogeneic blood SCT for children with Hurler's syndrome: results from the German multicenter approach MPS-HCT 2005. |
| Q46890992 | Allogeneic hematopoietic cell transplantation (HCT) in Hurler's syndrome using a reduced intensity preparative regimen |
| Q35903551 | Allogeneic stem cell transplantation for the treatment of lysosomal and peroxisomal metabolic diseases |
| Q55007551 | Alpha-Mannosidosis: Therapeutic Strategies. |
| Q30524999 | An exploratory study of brain function and structure in mucopolysaccharidosis type I: long term observations following hematopoietic cell transplantation (HCT) |
| Q47663948 | Assessments of neurocognitive and behavioral function in the mucopolysaccharidoses. |
| Q79552817 | Biochemical monitoring after haemopoietic stem cell transplant for Hurler syndrome (MPSIH): implications for functional outcome after transplant in metabolic disease |
| Q37330939 | Bone marrow transplantation for feline mucopolysaccharidosis I. |
| Q83171595 | Bone marrow transplantation in patients with storage diseases: a developing country experience |
| Q36236157 | Brain conditioning is instrumental for successful microglia reconstitution following hematopoietic stem cell transplantation |
| Q34501994 | Cardiac disease in mucopolysaccharidosis type I attributed to catecholaminergic and hemodynamic deficiencies |
| Q49557884 | Cell therapy for diverse central nervous system disorders: inherited metabolic diseases and autism |
| Q33692991 | Cell- and gene-based therapeutic approaches for neurological deficits in mucopolysaccharidoses |
| Q37649561 | Challenges in umbilical cord blood stem cell banking for stem cell reviews and reports |
| Q47434015 | Changes in the incidence, patterns and outcomes of graft failure following hematopoietic stem cell transplantation for Hurler syndrome |
| Q37758551 | Clinical aspects of neuropathic lysosomal storage disorders |
| Q35214507 | Clinical manifestations and treatment of mucopolysaccharidosis type I patients in Latin America as compared with the rest of the world |
| Q36009130 | Clinical outcomes following hematopoietic stem cell transplantation for the treatment of mucopolysaccharidosis VI |
| Q38768165 | Co-expression of MGMT(P140K) and alpha-L-iduronidase in primary hepatocytes from mucopolysaccharidosis type I mice enables efficient selection with metabolic correction |
| Q84074293 | Co-infusion of ex vivo-expanded, parental MSCs prevents life-threatening acute GVHD, but does not reduce the risk of graft failure in pediatric patients undergoing allogeneic umbilical cord blood transplantation |
| Q38785007 | Cognitive and adaptive measurement endpoints for clinical trials in mucopolysaccharidoses types I, II, and III: A review of the literature. |
| Q48331173 | Cognitive and neuroradiological improvement in three patients with attenuated MPS I treated by laronidase. |
| Q38787918 | Cognitive endpoints for therapy development for neuronopathic mucopolysaccharidoses: Results of a consensus procedure. |
| Q38765618 | Cognitive outcomes and age of detection of severe mucopolysaccharidosis type 1. |
| Q50856686 | Combination of enzyme replacement and hematopoietic stem cell transplantation as therapy for Hurler syndrome. |
| Q33974900 | Comparison of Endovascular and Intraventricular Gene Therapy With Adeno-Associated Virus-α-L-Iduronidase for Hurler Disease |
| Q34601968 | Concise review: umbilical cord blood transplantation: past, present, and future |
| Q35633105 | Cord blood in regenerative medicine: do we need immune suppression? |
| Q40210766 | Correction of clinical manifestations of canine mucopolysaccharidosis I with neonatal retroviral vector gene therapy |
| Q38081868 | Current and emerging management options for patients with Morquio A syndrome |
| Q36302871 | Current and emerging therapies for the lysosomal storage disorders |
| Q47668549 | Developmental and behavioral aspects of mucopolysaccharidoses with brain manifestations - Neurological signs and symptoms. |
| Q35559945 | Direct gene transfer to the CNS prevents emergence of neurologic disease in a murine model of mucopolysaccharidosis type I. |
| Q50354624 | Early treatment is associated with improved cognition in Hurler syndrome |
| Q45170206 | Effect of neonatal administration of a retroviral vector expressing alpha-L-iduronidase upon lysosomal storage in brain and other organs in mucopolysaccharidosis I mice |
| Q45861979 | Efficient correction of Fabry mice and patient cells mediated by lentiviral transduction of hematopoietic stem/progenitor cells |
| Q37051044 | Emerging trends in transplantation of inherited metabolic diseases |
| Q36637219 | Engineering a lysosomal enzyme with a derivative of receptor-binding domain of apoE enables delivery across the blood-brain barrier |
| Q38367272 | Enzyme replacement therapy in Hurler syndrome after failure of hematopoietic transplant |
| Q41105914 | Ethical and policy issues relating to progenitor-cell-based strategies for prevention of atherosclerosis |
| Q36759585 | Expanding the role of umbilical cord blood transplantation |
| Q45888388 | Expression in blood cells may contribute to biochemical and pathological improvements after neonatal intravenous gene therapy for mucopolysaccharidosis VII in dogs |
| Q37710965 | Feasibility of autologous cord blood cells for infants with hypoxic-ischemic encephalopathy |
| Q51651024 | Four successful pregnancies in a patient with mucopolysaccharidosis type I treated by allogeneic bone marrow transplantation. |
| Q48032820 | From cord to caudate: Characterizing umbilical cord blood stem cells and their paracrine interactions with the injured brain |
| Q42154353 | Functional abnormalities of heparan sulfate in mucopolysaccharidosis-I are associated with defective biologic activity of FGF-2 on human multipotent progenitor cells |
| Q37887166 | Gene and cell therapy for cystic fibrosis: from bench to bedside |
| Q30541387 | Gene therapy augments the efficacy of hematopoietic cell transplantation and fully corrects mucopolysaccharidosis type I phenotype in the mouse model. |
| Q36424472 | Gene therapy for lysosomal storage diseases. |
| Q34502978 | Gene therapy for lysosomal storage disorders: a good start |
| Q59340096 | Gene therapy for mucopolysaccharidoses: in vivo and ex vivo approaches |
| Q24631135 | Gene therapy for mucopolysaccharidosis |
| Q35362251 | Gene therapy for neurologic manifestations of mucopolysaccharidoses |
| Q35367599 | Glycosaminoglycan storage disorders: a review |
| Q82050079 | Graft failure following reduced-intensity cord blood transplantation for adult patients |
| Q51714921 | Growth and endocrine function in patients with Hurler syndrome after hematopoietic stem cell transplantation. |
| Q33918468 | Growth patterns and the use of growth hormone in the mucopolysaccharidoses |
| Q47395088 | Growth, final height and endocrine sequelae in a UK population of patients with Hurler syndrome (MPS1H). |
| Q47204887 | Haematopoietic cell transplantation (HCT) in combination with enzyme replacement therapy (ERT) in patients with Hurler syndrome. |
| Q34557967 | Hematopoietic differentiation of induced pluripotent stem cells from patients with mucopolysaccharidosis type I (Hurler syndrome) |
| Q30809579 | Hematopoietic stem cell transplantation for Morquio A syndrome |
| Q33418697 | Hematopoietic stem cell transplantation in infants |
| Q46807795 | High incidence of graft failure in unrelated cord blood transplantation using a reduced-intensity preparative regimen consisting of fludarabine and melphalan |
| Q51918509 | Hurler's syndrome: dental findings in a case treated with bone marrow transplantation in infancy. |
| Q64230256 | Immune cytopenia post-cord transplant in Hurler syndrome is a forme fruste of graft rejection |
| Q35319254 | Impact of enzyme replacement therapy and hematopoietic stem cell transplantation in patients with Morquio A syndrome. |
| Q38214093 | Implication of cord blood for cell-based therapy in refractory childhood diseases |
| Q30493396 | Improved retroviral vector design results in sustained expression after adult gene therapy in mucopolysaccharidosis I mice |
| Q45866168 | Improvements in mucopolysaccharidosis I mice after adult retroviral vector-mediated gene therapy with immunomodulation. |
| Q42273263 | Increased longevity and metabolic correction following syngeneic BMT in a murine model of mucopolysaccharidosis type I. |
| Q34054239 | Innovative therapy for Classic Galactosemia - tale of two HTS. |
| Q48585796 | Intracerebroventricular transplantation of human bone marrow-derived multipotent progenitor cells in an immunodeficient mouse model of mucopolysaccharidosis type I (MPS-I). |
| Q45856196 | Intrathecal administration of AAV vectors for the treatment of lysosomal storage in the brains of MPS I mice |
| Q30497736 | Intrathecal enzyme replacement therapy: successful treatment of brain disease via the cerebrospinal fluid |
| Q35007847 | Intravenous administration of auto serum-expanded autologous mesenchymal stem cells in stroke |
| Q47926676 | Intravenous administration of human umbilical cord blood cells in an animal model of MPS III B. |
| Q38741428 | Liver-Directed Human Amniotic Epithelial Cell Transplantation Improves Systemic Disease Phenotype in Hurler Syndrome Mouse Model |
| Q48358275 | Long-term efficacy of hematopoietic stem cell transplantation on brain involvement in patients with mucopolysaccharidosis type II: a nationwide survey in Japan |
| Q34318553 | Long-term follow-up after gene therapy for canavan disease. |
| Q35220724 | Long-term functional outcomes of children with hurler syndrome treated with unrelated umbilical cord blood transplantation |
| Q34938821 | Long-term therapeutic efficacy of allogenic bone marrow transplantation in a patient with mucopolysaccharidosis IVA. |
| Q37401176 | Lysosomal storage disorders in the newborn |
| Q37296284 | Mankind's first natural stem cell transplant. |
| Q37020116 | Mesenchymal stem cells derived from peripheral blood protects against ischemia. |
| Q34870734 | Metachromatic leukodystrophy: an overview of current and prospective treatments |
| Q37109605 | Methods for assessing neurodevelopment in lysosomal storage diseases and related disorders: a multidisciplinary perspective |
| Q33947305 | Methods of neurodevelopmental assessment in children with neurodegenerative disease: Sanfilippo syndrome |
| Q35104361 | Molecular Genetics and Metabolism Report Long-term follow-up of post hematopoietic stem cell transplantation for Hurler syndrome: clinical, biochemical, and pathological improvements |
| Q39067954 | Mortality after hematopoietic stem cell transplantation for severe mucopolysaccharidosis type I: the 30-year University of Minnesota experience |
| Q36894699 | Mucopolysaccharidosis (MPS) Physical Symptom Score: Development, Reliability, and Validity |
| Q45858291 | Mucopolysaccharidosis I cats mount a cytotoxic T lymphocyte response after neonatal gene therapy that can be blocked with CTLA4-Ig. |
| Q34562817 | Mucopolysaccharidosis I, II, and VI: Brief review and guidelines for treatment. |
| Q39160586 | Mucopolysaccharidosis I: Alpha-L-Iduronidase mutations in three Tunisian families |
| Q35153234 | Neonatal bone marrow transplantation prevents bone pathology in a mouse model of mucopolysaccharidosis type I. |
| Q36582259 | Neonatal cellular and gene therapies for mucopolysaccharidoses: the earlier the better? |
| Q40663776 | Neonatal tolerance induction enables accurate evaluation of gene therapy for MPS I in a canine model |
| Q41533871 | Neonatal umbilical cord blood transplantation halts skeletal disease progression in the murine model of MPS-I. |
| Q36748869 | Neurodegeneration and cell replacement. |
| Q36646889 | New therapeutic options for lysosomal storage disorders: enzyme replacement, small molecules and gene therapy |
| Q83116248 | Newborn screening for lysosomal storage disorders |
| Q37763610 | Newborn screening for neuropathic lysosomal storage disorders. |
| Q35600316 | Normalization and improvement of CNS deficits in mice with Hurler syndrome after long-term peripheral delivery of BBB-targeted iduronidase |
| Q36626146 | Novel cell therapy approaches for brain repair |
| Q33804960 | Open issues in Mucopolysaccharidosis type I-Hurler |
| Q49411650 | Ophthalmologic Findings in Patients with Neuro-metabolic Disorders |
| Q37020064 | Optimization of a therapeutic protocol for intravenous injection of human mesenchymal stem cells after cerebral ischemia in adult rats |
| Q64274669 | Oral manifestation and root canal therapy of the patient with mucopolysaccharidosis |
| Q37972587 | Orthopaedic aspects of mucopolysaccharidoses |
| Q27690888 | Orthopaedic management of Hurler's disease after hematopoietic stem cell transplantation: a systematic review. |
| Q33449843 | Outcomes of 122 diverse adult and pediatric cord blood transplant recipients from a large cord blood bank |
| Q80387211 | Outcomes of hematopoietic stem cell transplantation for Hurler's syndrome in Europe: a risk factor analysis for graft failure |
| Q34333060 | Outcomes of transplantation using various hematopoietic cell sources in children with Hurler syndrome after myeloablative conditioning |
| Q38746020 | Paracrine molecules of mesenchymal stem cells for hematopoietic stem cell niche |
| Q51800411 | Pre-transplant risk factors affecting outcome in Hurler syndrome. |
| Q42120275 | Precocious initiation of spermatogenesis in a 19-month-old boy with Hurler syndrome |
| Q30491775 | Progression of multiple behavioral deficits with various ages of onset in a murine model of Hurler syndrome. |
| Q33579453 | Radiographic evaluation of bones and joints in mucopolysaccharidosis I and VII dogs after neonatal gene therapy |
| Q93209008 | Recent trends in mucopolysaccharidosis research |
| Q57216387 | Reduced intensity allogeneic umbilical cord blood transplantation in children and adolescent recipients with malignant and non-malignant diseases |
| Q37446490 | Reprogramming erythroid cells for lysosomal enzyme production leads to visceral and CNS cross-correction in mice with Hurler syndrome |
| Q34579526 | Research challenges in central nervous system manifestations of inborn errors of metabolism |
| Q46745267 | Response to the letter: allogeneic hematopoietic stem cell transplantation for treatment of Gaucher disease |
| Q30532642 | Retroviral-vector-mediated gene therapy to mucopolysaccharidosis I mice improves sensorimotor impairments and other behavioral deficits |
| Q83299926 | Rheumatologic aspects of lysosomal storage diseases |
| Q47848350 | Safety and efficacy of enzyme replacement therapy in combination with hematopoietic stem cell transplantation in Hurler syndrome |
| Q36013774 | Safety and feasibility of countering neurological impairment by intravenous administration of autologous cord blood in cerebral palsy |
| Q37130107 | Sanfilippo syndrome: a mini-review |
| Q37033846 | Searching for alternative hematopoietic stem cell donors for pediatric patients |
| Q82662038 | Severe regimen-related toxicity of second transplantation for graft failure following reduced-intensity cord blood transplantation in an adult patient |
| Q34763337 | Short-term growth hormone treatment in children with Hurler syndrome after hematopoietic cell transplantation |
| Q45878694 | Skeletal response to lentiviral mediated gene therapy in a mouse model of MPS VII. |
| Q84435258 | Stem cell biobanks |
| Q37053159 | Stem cells and neurological diseases. |
| Q37962831 | Stem cells as a therapeutic approach to chronic kidney diseases |
| Q37292534 | Supporting family adaptation to presymptomatic and "untreatable" conditions in an era of expanded newborn screening |
| Q86654372 | TCRαβ CD19 depletion in allogeneic haematopoietic stem cell transplantation performed for Hurler syndrome |
| Q36050021 | The Cord Blood Apgar: a novel scoring system to optimize selection of banked cord blood grafts for transplantation (CME). |
| Q79870081 | The MPS I registry: design, methodology, and early findings of a global disease registry for monitoring patients with Mucopolysaccharidosis Type I |
| Q36602806 | The Neurobehavioral Phenotype in Mucopolysaccharidosis Type IIIB: an Exploratory Study |
| Q36336608 | The Role of HLA in Cord Blood Transplantation. |
| Q81434774 | The controversial and changing role of haematopoietic cell transplantation for lysosomal storage disorders: an update |
| Q51886338 | The craniocervical junction following successful haematopoietic stem cell transplantation for mucopolysaccharidosis type I H (Hurler syndrome). |
| Q43600333 | The effects of early and late bone marrow transplantation in siblings with alpha-mannosidosis. Is early haematopoietic cell transplantation the preferred treatment option? |
| Q36082084 | The first 5 years of clinical experience with laronidase enzyme replacement therapy for mucopolysaccharidosis I. |
| Q37059193 | The mucopolysaccharidoses: a success of molecular medicine. |
| Q37950844 | The role of enzyme replacement therapy in severe Hunter syndrome-an expert panel consensus |
| Q34038205 | The role of microglia in human disease: therapeutic tool or target? |
| Q26852308 | Therapies for the bone in mucopolysaccharidoses |
| Q37972592 | Therapy for the mucopolysaccharidoses |
| Q37788417 | Tissue regeneration potential in human umbilical cord blood |
| Q43730176 | Topical issues in unrelated donor haematopoietic stem cell transplants: a report from a workshop convened by the Anthony Nolan Trust in London - 2005. |
| Q39151620 | Transplantation in inborn errors of metabolism: current considerations and future perspectives |
| Q45174143 | Transplantation of allogeneic CD34-selected stem cells after fludarabine-based conditioning regimen for children with mucopolysaccharidosis 1H (M. Hurler). |
| Q45874710 | Treatment of brain disease in the mucopolysaccharidoses |
| Q37366102 | Treatment of hip dysplasia in patients with mucopolysaccharidosis type I after hematopoietic stem cell transplantation: results of an international consensus procedure |
| Q33502511 | Treatment reduces or stabilizes brain imaging abnormalities in patients with MPS I and II. |
| Q36501354 | Trends in haematopoietic cell transplantation for inborn errors of metabolism |
| Q36272165 | Twenty years of progress in biogerontology research |
| Q79609998 | Umbilical cord blood banking options and the prenatal patient: an obstetrician's perspective |
| Q38510475 | Umbilical cord blood donation: public or private? |
| Q37607972 | Umbilical cord blood transplantation for non-malignant diseases |
| Q36167454 | Umbilical cord blood-derived stem cells and brain repair. |
| Q40214372 | Unrelated CD3/CD19-depleted peripheral stem cell transplantation for Hurler syndrome |
| Q36915945 | Unrelated donor umbilical cord blood transplantation for inherited metabolic disorders in 159 pediatric patients from a single center: influence of cellular composition of the graft on transplantation outcomes. |
| Q34048540 | Untying the Gordian knot: policies, practices, and ethical issues related to banking of umbilical cord blood |
| Q36928836 | Update on umbilical cord blood transplantation |
| Q35086582 | Use of a precious resource: parental decision making about using autologous umbilical cord blood in studies involving young children with type 1 diabetes |
| Q50790997 | Variable disease progression after successful stem cell transplantation: prospective follow-up investigations in eight patients with Hurler syndrome. |
| Q30541880 | Whole body correction of mucopolysaccharidosis IIIA by intracerebrospinal fluid gene therapy |
| Q64047006 | [Gaucher disease, Fabry disease and mucopolysaccharidosis type I--how can the rheumatologist recognise these patients?] |
| Q53451923 | [The importance of the use of stem cells for public health]. |
| Q39952328 | alpha-L-iduronidase therapy for mucopolysaccharidosis type I. |
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