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Adrenoleukodystrophy

  • Childhood Cerebral X-Linked Adrenoleukodystrophy with Atypical Neuroimaging Abnormalities and a No…

    Childhood Cerebral X-Linked Adrenoleukodystrophy with Atypical Neuroimaging Abnormalities and a No…

  • Nicole Trask, Pharmd, Planning for the 2019 Specialty Drug Spend

    Nicole Trask, Pharmd, Planning for the 2019 Specialty Drug Spend

  • Rett Syndrome: Coming to Terms with Treatment

    Rett Syndrome: Coming to Terms with Treatment

  • Pelizaeus-Merzbacher Disease (Pmd)

    Pelizaeus-Merzbacher Disease (Pmd)

  • X-Linked Diseases: Susceptible Females

    X-Linked Diseases: Susceptible Females

  • X Linked Adrenoleukodystrophy: Clinical Presentation, Diagnosis, and Therapy

    X Linked Adrenoleukodystrophy: Clinical Presentation, Diagnosis, and Therapy

  • Page 1 of 303 2020 Basic PDP Prior Authorization Criteria Effective 12/01/2020

    Page 1 of 303 2020 Basic PDP Prior Authorization Criteria Effective 12/01/2020

  • Transporters

    Transporters

  • Estimating the Financial Impact of Gene Therapy*

    Estimating the Financial Impact of Gene Therapy*

  • New Diagnostic Criteria for Infantile Nystagmus. an Upgraded

    New Diagnostic Criteria for Infantile Nystagmus. an Upgraded

  • Leukodystrophies by Raphael Schiffmann MD (Dr

    Leukodystrophies by Raphael Schiffmann MD (Dr

  • Adeno-Associated Virus 8-Mediated Gene Therapy for Choroideremia: Preclinical Studies in in Vitro Ind in Vivo Models

    Adeno-Associated Virus 8-Mediated Gene Therapy for Choroideremia: Preclinical Studies in in Vitro Ind in Vivo Models

  • Progressive Spastic Paraparesis in a Young Male

    Progressive Spastic Paraparesis in a Young Male

  • Outcomes After Allogeneic Hematopoietic Cell Transplantation for Childhood Cerebral Adrenoleukodystrophy: the Largest Single-Institution Cohort Report

    Outcomes After Allogeneic Hematopoietic Cell Transplantation for Childhood Cerebral Adrenoleukodystrophy: the Largest Single-Institution Cohort Report

  • Loss of the Sphingolipid Desaturase DEGS1 Causes Hypomyelinating Leukodystrophy

    Loss of the Sphingolipid Desaturase DEGS1 Causes Hypomyelinating Leukodystrophy

  • (CHMP) Agenda for the Meeting on 17-20 May 2021 Chair: Harald Enzmann – Vice-Chair: Bruno Sepodes

    (CHMP) Agenda for the Meeting on 17-20 May 2021 Chair: Harald Enzmann – Vice-Chair: Bruno Sepodes

  • Hematopoietic Stem Cell-Based Gene Therapy Clinical Impact and Current Challenges

    Hematopoietic Stem Cell-Based Gene Therapy Clinical Impact and Current Challenges

  • X-Linked Adrenoleukodystrophy Presenting with Positional Downbeat Nystagmus

    X-Linked Adrenoleukodystrophy Presenting with Positional Downbeat Nystagmus

Top View
  • Using Patientderived Cells to Model Xlinked Adrenoleukodystrophy
  • High Connectivity Between Reduced Cortical Thickness and Disrupted White Matter Tracts in Long-Standing Type 1 Diabetes Daniel T
  • A Case of Adrenoleukodystrophy Presenting As Progressive Cerebellar Dysfunction
  • Multiple Sclerosis
  • Towards Mechanism- Based Treatments for Fragile X Syndrome
  • ( 12 ) United States Patent
  • Adrenoleukodystrophy in the Differential Diagnosis of Boys Presenting with Primary Adrenal Insufficiency Without Adrenal Antibodies
  • Preimplantation Genetic Diagnosis of X-Linked Adrenoleukodystrophy with Gender Determination Using Multiple Displacement Amplification
  • Rare Forms of Dementia
  • The Leukodystrophies
  • Long-Term Outcome of Schilder Disease Treated with Interferon-B Wei-Sheng Lin, MD,A Meng-Fai Kuo, MD, Phd,B Steven Shinn-Forng Peng, MD, Phd,C Pi-Chuan Fan, MD, Phdd
  • X-Linked Adrenoleukodystrophy Presenting As Autosomal Dominant Pure Hereditary Spastic Paraparesis C J Shaw-Smith, S J G Lewis, E Reid
  • Adrenoleukodystrophy with Frontal Lobe Involvement and Attention
  • Multiple Sclerosis: Implications for Practice
  • Emerging Therapies Workgroup (June 18, 2019)
  • Familial Spinocerebellar Degeneration As an Expression of Adrenoleukodystrophy
  • Positive XL Female
  • Blueprint Genetics My Retina Tracker Program Panel


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