Search Results - "lysosomal storage disorders"

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  1. 21
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    GCase Enhancers: A Potential Therapeutic Option for Gaucher Disease and Other Neurological Disorders by Macarena Martínez-Bailén, Francesca Clemente, Camilla Matassini, Francesca Cardona

    Published 2022
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  2. 22
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    IgG Fusion Proteins for Brain Delivery of Biologics via Blood-Brain Barrier Receptor-Mediated Transport by Ruben J. Boado

    Published 2022
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  3. 23
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    Molecular Basis of Metachromatic Leukodystrophies by J Gordon Millichap

    Published 1991
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  4. 24
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    First person - Lena Marie Westermann

    Published 2020
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  5. 25
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    The Complexity of Pain Management in Children Affected by Mucopolysaccharidoses by Sabrina Congedi, Chiara Di Pede, Maurizio Scarpa, Angelica Rampazzo, Franca Benini

    Published 2017
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  6. 26
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    Newborn Screening for Lysosomal Disease: Mission Creep and a Taste of Things to Come? by Bridget Wilcken

    Published 2018
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  7. 27
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    The new frame for Mucopolysaccharidoses by Rossella Parini, Andrea Biondi

    Published 2018
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  8. 28
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    Ocular features in mucopolysaccharidosis: diagnosis and treatment by Alessandra Del Longo, Elena Piozzi, Fiammetta Schweizer

    Published 2018
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  9. 29
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    Coenzyme Q10: Role in Less Common Age-Related Disorders by David Mantle, Iain P. Hargreaves

    Published 2022
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  10. 30
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    FARBER DISEASE - DISEASE DESCRIPTION WITH CASE REPORTS by I. A. Chikova, N. V. Buchinskaya, М. М. Kostik, V. V. Avramenko, O. L. Krasnogorskaya, R. A. Nasirov, T. Levade, V. G. Chasnyk

    Published 2014
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  11. 31
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    Neuronopathic Types of Mucopolysaccharidoses: Pathogenesis and Emerging Treatments by L. A. Osipova, L. M. Kuzenkova, L. S. Namazova-Baranova, A. K. Gevorkyan, T. V. Podkletnova, N. D. Vashakmadze

    Published 2015
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  12. 32
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    Niemann-Pick Disease Type C (NPDC) by Mutation of <i>NPC1</i> and <i>NPC2</i>: Aberrant Lysosomal Cholesterol Trafficking and Oxidative Stress by Dongun Lee, Jeong Hee Hong

    Published 2023
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  13. 33
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    Gene therapy for mucopolysaccharidoses: in vivo and ex vivo approaches by Alessandro Fraldi, Marta Serafini, Nicolina Cristina Sorrentino, Bernhard Gentner, Alessandro Aiuti, Maria Ester Bernardo

    Published 2018
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  14. 34
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    Pediatric Sleep Respiratory Disorders: A Narrative Review of Epidemiology and Risk Factors by Marta Piotto, Antonella Gambadauro, Alessia Rocchi, Mara Lelii, Barbara Madini, Lucia Cerrato, Federica Chironi, Youssra Belhaj, Maria Francesca Patria

    Published 2023
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  15. 35
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    Gene therapy for rare haematological and neurometabolic paediatric diseases by Vera Gallo, Alessandro Aiuti

    Published 2024
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  16. 36
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    First hematopoietic stem cell transplantation in a child with late infantile Krabbe disease in Poland by Izabella Miśkiewicz-Migoń, Justyna Miśkiewicz-Bujna, Monika Mielcarek-Siedziuk, Jowita Frączkiewicz, Ewa Gorczyńska, Krzysztof Kałwak

    Published 2021
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  17. 37
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    Current Practices for U.S. Newborn Screening of Pompe Disease and MPSI by Elizabeth G. Ames, Rachel Fisher, Mary Kleyn, Ayesha Ahmad

    Published 2020
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  18. 38
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    Individual Treatment Trials-Do Experts Know and Use This Option to Improve the Treatability of Mucopolysaccharidosis? by Anna-Maria Wiesinger, Hannah Strobl, Florian B. Lagler

    Published 2023
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  19. 39
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    Placental Findings in Lysosomal Storage Disease Diagnosis: A Case Report of Galactosialidosis by Sasha Libbrecht, Francois Eyskens, Sabine Declercq, Cecile Colpaert

    Published 2020
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  20. 40
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    Neonatal Screening for MPS Disorders in Latin America: A Survey of Pilot Initiatives by Francyne Kubaski, Inês Sousa, Tatiana Amorim, Danilo Pereira, Joe Trometer, Alexandre Souza, Enzo Ranieri, Giulia Polo, Alberto Burlina, Ana Carolina Brusius-Facchin, Alice Brinckmann Oliveira Netto, Shunji Tomatsu, Roberto Giugliani

    Published 2020
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