Search Results - Finkel, Richard S.

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  1. 1
    Readthrough Strategies for Suppression of Nonsense Mutations in Duchenne/Becker Muscular Dystrophy: Aminoglycosides and Ataluren (PTC124)

    Readthrough Strategies for Suppression of Nonsense Mutations in Duchenne/Becker Muscular Dystrophy: Aminoglycosides and Ataluren (PTC124) by Finkel, Richard S.

    Published 2010
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  2. 2
    Widening gap in age at muscular dystrophy–associated death between blacks and whites, 1986–2005

    Widening gap in age at muscular dystrophy–associated death between blacks and whites, 1986–2005 by Kenneson, Aileen, Vatave, Ajay, Finkel, Richard

    Published 2010
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  3. 3
    Editorial: Spinal Muscular Atrophy: Evolutions and Revolutions of Modern Therapy

    Editorial: Spinal Muscular Atrophy: Evolutions and Revolutions of Modern Therapy by Finkel, Richard S., Schara-Schmidt, Ulrike, Hagenacker, Tim

    Published 2020
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  4. 4
    Spinal Muscular Atrophy Type I: Is It Ethical to Standardize Supportive Care Intervention in Clinical Trials?

    Spinal Muscular Atrophy Type I: Is It Ethical to Standardize Supportive Care Intervention in Clinical Trials? by Finkel, Richard S., Bishop, Kathie M., Nelson, Robert M.

    Published 2016
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  5. 5
    Unusual Cardiac “Masses” in a Newborn with Infantile Pompe Disease

    Unusual Cardiac “Masses” in a Newborn with Infantile Pompe Disease by Swarr, Daniel T., Kaufman, Beth, Fogel, Mark A., Finkel, Richard, Ganesh, Jaya

    Published 2011
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  6. 6
    Evolution of Next Generation Therapeutics: Past, Present, and Future of Precision Medicines

    Evolution of Next Generation Therapeutics: Past, Present, and Future of Precision Medicines by Ramamoorthy, Anuradha, Karnes, Jason H., Finkel, Richard, Blanchard, Rebecca, Pacanowski, Michael

    Published 2019
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  7. 7
    The Use of Autologous Blood Patch in Ullrich Muscular Dystrophy and Recurrent Pneumothorax

    The Use of Autologous Blood Patch in Ullrich Muscular Dystrophy and Recurrent Pneumothorax by Navas Nazario, Aledie, Cooper, Felicia I, Weber-Guzman, Fabiola, Finkel, Richard S

    Published 2022
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  8. 8
    Umbilical cord blood transplantation for juvenile metachromatic leukodystrophy

    Umbilical cord blood transplantation for juvenile metachromatic leukodystrophy by Pierson, Tyler Mark, Bonnemann, Carsten G, Finkel, Richard S, Bunin, Nancy, Tennekoon, Gihan I

    Published 2008
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  9. 9
    SMA-EUROPE workshop report: opportunities and challenges in developing clinical trials for spinal muscular atrophy in Europe

    SMA-EUROPE workshop report: opportunities and challenges in developing clinical trials for spinal muscular atrophy in Europe by Kayadjanian, Nathalie, Burghes, Arthur, Finkel, Richard S, Mercuri, Eugenio, Rouault, Francoise, Schwersenz, Inge, Talbot, Kevin

    Published 2013
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  10. 10
    Serial Casting for the Management of Ankle Contracture in Duchenne Muscular Dystrophy

    Serial Casting for the Management of Ankle Contracture in Duchenne Muscular Dystrophy by Glanzman, Allan M., Flickinger, Jean M., Dholakia, Kripa H., Bönnemann, Carsten G., Finkel, Richard S.

    Published 2011
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  11. 11
    UNRELATED UMBILICAL CORD BLOOD TRANSPLANT FOR JUVENILE METACHROMATIC LEUKODYSTROPHY: A FIVE-YEAR FOLLOW-UP IN THREE AFFECTED SIBLINGS

    UNRELATED UMBILICAL CORD BLOOD TRANSPLANT FOR JUVENILE METACHROMATIC LEUKODYSTROPHY: A FIVE-YEAR FOLLOW-UP IN THREE AFFECTED SIBLINGS by Cable, Casey, Finkel, Richard S, Lehky, Tanya J, Biassou, Nadia M, Wiggs, Edythe A, Bunin, Nancy, Pierson, Tyler Mark

    Published 2010
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  12. 12
    GFPT1-myasthenia: Clinical, structural, and electrophysiologic heterogeneity

    GFPT1-myasthenia: Clinical, structural, and electrophysiologic heterogeneity by Selcen, Duygu, Shen, Xin-Ming, Milone, Margherita, Brengman, Joan, Ohno, Kinji, Deymeer, Feza, Finkel, Richard, Rowin, Julie, Engel, Andrew G.

    Published 2013
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  13. 13
    Spectrum of Neuropathophysiology in Spinal Muscular Atrophy Type I

    Spectrum of Neuropathophysiology in Spinal Muscular Atrophy Type I by Harding, Brian N., Kariya, Shingo, Monani, Umrao R., Chung, Wendy K., Benton, Maryjane, Yum, Sabrina W., Tennekoon, Gihan, Finkel, Richard S.

    Published 2015
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  14. 14
    An Integrated Safety Analysis of Infants and Children with Symptomatic Spinal Muscular Atrophy (SMA) Treated with Nusinersen in Seven Clinical Trials

    An Integrated Safety Analysis of Infants and Children with Symptomatic Spinal Muscular Atrophy (SMA) Treated with Nusinersen in Seven Clinical Trials by Darras, Basil T., Farrar, Michelle A., Mercuri, Eugenio, Finkel, Richard S., Foster, Richard, Hughes, Steven G., Bhan, Ishir, Farwell, Wildon, Gheuens, Sarah

    Published 2019
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  15. 15
    MRI/MRS Evaluation of a Female Carrier of Duchenne Muscular Dystrophy

    MRI/MRS Evaluation of a Female Carrier of Duchenne Muscular Dystrophy by Forbes, Sean C., Lott, Donovan J., Finkel, Richard S., Senesac, Claudia, Byrne, Barry J., Sweeney, H. L., Walter, Glenn A., Vandenborne, Krista

    Published 2012
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  16. 16
    Adeno-associated virus serotype 9 antibodies in patients screened for treatment with onasemnogene abeparvovec

    Adeno-associated virus serotype 9 antibodies in patients screened for treatment with onasemnogene abeparvovec by Day, John W., Finkel, Richard S., Mercuri, Eugenio, Swoboda, Kathryn J., Menier, Melissa, van Olden, Rudolf, Tauscher-Wisniewski, Sitra, Mendell, Jerry R.

    Published 2021
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  17. 17
    Motor function in type 2 and 3 SMA patients treated with Nusinersen: a critical review and meta-analysis

    Motor function in type 2 and 3 SMA patients treated with Nusinersen: a critical review and meta-analysis by Coratti, Giorgia, Cutrona, Costanza, Pera, Maria Carmela, Bovis, Francesca, Ponzano, Marta, Chieppa, Fabrizia, Antonaci, Laura, Sansone, Valeria, Finkel, Richard, Pane, Marika, Mercuri, Eugenio

    Published 2021
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  18. 18
    Restoration of Nusinersen Levels Following Treatment Interruption in People With Spinal Muscular Atrophy: Simulations Based on a Population Pharmacokinetic Model

    Restoration of Nusinersen Levels Following Treatment Interruption in People With Spinal Muscular Atrophy: Simulations Based on a Population Pharmacokinetic Model by MacCannell, Drew, Berger, Zdenek, Kirschner, Janbernd, Mercuri, Eugenio, Farrar, Michelle A., Iannaccone, Susan T., Kuntz, Nancy L., Finkel, Richard S., Valente, Marta, Muntoni, Francesco

    Published 2022
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  19. 19
    Distribution of weight, stature, and growth status in children and adolescents with spinal muscular atrophy: An observational retrospective study in the United States

    Distribution of weight, stature, and growth status in children and adolescents with spinal muscular atrophy: An observational retrospective study in the United States by Darras, Basil T., Guye, Sabrina, Hoffart, Janine, Schneider, Sophie, Gravestock, Isaac, Gorni, Ksenija, Fuerst‐Recktenwald, Sabine, Scalco, Renata S., Finkel, Richard S., De Vivo, Darryl C.

    Published 2022
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  20. 20
    The impact of antibodies on clinical outcomes in diseases treated with therapeutic protein: Lessons learned from infantile Pompe disease

    The impact of antibodies on clinical outcomes in diseases treated with therapeutic protein: Lessons learned from infantile Pompe disease by Banugaria, Suhrad G., Prater, Sean N., Ng, Yiu-Ki, Kobori, Joyce A., Finkel, Richard S., Ladda, Roger L., Chen, Yuan-Tsong, Rosenberg, Amy S., Kishnani, Priya S.

    Published 2011
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