Search Results - "Parsons, Julie A."

Nusinersen initiated in infants during the presymptomatic stage of spinal muscular atrophy: Interim efficacy and safety results from the Phase 2 NURTURE study by De Vivo, Darryl C., Bertini, Enrico, Swoboda, Kathryn J., Hwu, Wuh-Liang, Crawford, Thomas O., Finkel, Richard S., Kirschner, Janbernd, Kuntz, Nancy L., Parsons, Julie A., Ryan, Monique M., Butterfield, Russell J., Topaloglu, Haluk, Ben-Omran, Tawfeg, Sansone, Valeria A., Jong, Yuh-Jyh, Shu, Francy, Staropoli, John F., Kerr, Douglas, Sandrock, Alfred W., Stebbins, Christopher, Petrillo, Marco, Braley, Gabriel, Johnson, Kristina, Foster, Richard, Gheuens, Sarah, Bhan, Ishir, Reyna, Sandra P., Fradette, Stephanie, Farwell, Wildon

“…•NURTURE is an ongoing study of nusinersen started in a presymptomatic stage of SMA.•All infants were ≥25 months old, and alive without permanent…”
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Subacute Liver Failure Following Gene Replacement Therapy for Spinal Muscular Atrophy Type 1 by Feldman, Amy G., Parsons, Julie A., Dutmer, Cullen M., Veerapandiyan, Aravindhan, Hafberg, Einar, Maloney, Nolan, Mack, Cara L.

“…Spinal muscular atrophy is a neurodegenerative disease resulting from irreversible loss of anterior horn cells owing to biallelic deletions/mutations in the…”
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Medical management of muscle weakness in Duchenne muscular dystrophy by Rivera, Sarah R, Jhamb, Sumit K, Abdel-Hamid, Hoda Z, Acsadi, Gyula, Brandsema, John, Ciafaloni, Emma, Darras, Basil T, Iannaccone, Susan T, Konersman, Chamindra G, Kuntz, Nancy L, McDonald, Craig M, Parsons, Julie A, Tesi Rocha, Carolina, Zaidman, Craig M, Butterfield, Russell J, Connolly, Anne M, Mathews, Katherine D

“…Duchenne muscular dystrophy (DMD) is a childhood onset muscular dystrophy leading to shortened life expectancy. There are gaps in published DMD care guidelines…”
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Long-Term Follow-Up Cares and Check Initiative: A Program to Advance Long-Term Follow-Up in Newborns Identified with a Disease through Newborn Screening by Lietsch, Mei, Chan, Kee, Taylor, Jennifer, Lee, Bo Hoon, Ciafaloni, Emma, Kwon, Jennifer M, Waldrop, Megan A, Butterfield, Russell J, Rathore, Geetanjali, Veerapandiyan, Aravindhan, Kapil, Arya, Parsons, Julie A, Gibbons, Melissa, Brower, Amy

“…In the United States and around the world, newborns are screened on a population basis for conditions benefiting from pre-symptomatic diagnosis and treatment…”
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Onasemnogene abeparvovec for presymptomatic infants with two copies of SMN2 at risk for spinal muscular atrophy type 1: the Phase III SPR1NT trial by Strauss, Kevin A., Farrar, Michelle A., Muntoni, Francesco, Saito, Kayoko, Mendell, Jerry R., Servais, Laurent, McMillan, Hugh J., Finkel, Richard S., Swoboda, Kathryn J., Kwon, Jennifer M., Zaidman, Craig M., Chiriboga, Claudia A., Iannaccone, Susan T., Krueger, Jena M., Parsons, Julie A., Shieh, Perry B., Kavanagh, Sarah, Tauscher-Wisniewski, Sitra, McGill, Bryan E., Macek, Thomas A.

“…SPR1NT ( NCT03505099 ) was a Phase III, multicenter, single-arm study to investigate the efficacy and safety of onasemnogene abeparvovec for presymptomatic…”
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Onasemnogene abeparvovec for presymptomatic infants with three copies of SMN2 at risk for spinal muscular atrophy: the Phase III SPR1NT trial by Strauss, Kevin A., Farrar, Michelle A., Muntoni, Francesco, Saito, Kayoko, Mendell, Jerry R., Servais, Laurent, McMillan, Hugh J., Finkel, Richard S., Swoboda, Kathryn J., Kwon, Jennifer M., Zaidman, Craig M., Chiriboga, Claudia A., Iannaccone, Susan T., Krueger, Jena M., Parsons, Julie A., Shieh, Perry B., Kavanagh, Sarah, Wigderson, Melissa, Tauscher-Wisniewski, Sitra, McGill, Bryan E., Macek, Thomas A.

“…Most children with biallelic SMN1 deletions and three SMN2 copies develop spinal muscular atrophy (SMA) type 2. SPR1NT ( NCT03505099 ), a Phase III,…”
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The care of patients with Duchenne, Becker, and other muscular dystrophies in the COVID‐19 pandemic by Veerapandiyan, Aravindhan, Wagner, Kathryn R., Apkon, Susan, McDonald, Craig M., Mathews, Katherine D., Parsons, Julie A., Wong, Brenda L., Eichinger, Katy, Shieh, Perry B., Butterfield, Russell J., Rao, Vamshi K., Smith, Edward C., Proud, Crystal M., Connolly, Anne M., Ciafaloni, Emma

“…The coronavirus disease 2019 (COVID‐19) pandemic has resulted in the reorganization of health‐care settings affecting clinical care delivery to patients with…”
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Continued benefit of nusinersen initiated in the presymptomatic stage of spinal muscular atrophy: 5‐year update of the NURTURE study by Crawford, Thomas O., Swoboda, Kathryn J., De Vivo, Darryl C., Bertini, Enrico, Hwu, Wuh‐Liang, Finkel, Richard S., Kirschner, Janbernd, Kuntz, Nancy L., Nazario, Aledie Navas, Parsons, Julie A., Pechmann, Astrid, Ryan, Monique M., Butterfield, Russell J., Topaloglu, Haluk, Ben‐Omran, Tawfeg, Sansone, Valeria A., Jong, Yuh‐Jyh, Shu, Francy, Zhu, Cong, Raynaud, Stephanie, Lago, Tiffany R., Paradis, Angela D., Foster, Richard, Chin, Russell, Berger, Zdenek

“…Introduction/Aims NURTURE (NCT02386553) is an open‐label study of nusinersen in children (two SMN2 copies, n = 15; three SMN2 copies, n = 10) who initiated…”
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A retrospective cohort study of children with spinal muscular atrophy type 2 receiving anesthesia for intrathecal administration of nusinersen by Bielsky, Alan R., Fuhr, Peter G., Parsons, Julie A., Yaster, Myron

“…Summary Introduction Spinal muscular atrophy is characterized by loss of motor neurons in the anterior horn of the spinal cord with resultant proximal muscle…”
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Spinal muscular atrophy care in the COVID‐19 pandemic era by Veerapandiyan, Aravindhan, Connolly, Anne M., Finkel, Richard S., Arya, Kapil, Mathews, Katherine D., Smith, Edward C., Castro, Diana, Butterfield, Russell J., Parsons, Julie A., Servais, Laurent, Kuntz, Nancy, Rao, Vamshi K., Brandsema, John F., Mercuri, Eugenio, Ciafaloni, Emma

“…The coronavirus disease 2019 (COVID‐19) pandemic has resulted in reorganization of healthcare settings affecting the delivery of clinical care to patients with…”
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How to Kiss and Make-Up (or Not!): Postconflict Behavior and Affective Recovery From Conflict by Parsons, Julie A., Prager, Karen J., Wu, Sining, Poucher, Jesse W., Hansen, Marissa Pullum, Shirvani, Forouz

“…We designed 3 studies to identify postconflict behaviors that aid or hinder couple partners' emotional recoveries from their conflicts. For Study 1, we created…”
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Outcomes for patients in the RESTORE registry with spinal muscular atrophy and four or more SMN2 gene copies treated with onasemnogene abeparvovec by Tizzano, Eduardo F., Quijano-Roy, Susana, Servais, Laurent, Parsons, Julie A., Aharoni, Sharon, Lakhotia, Arpita, Finkel, Richard S., Mercuri, Eugenio, Kirschner, Janbernd, De Vivo, Darryl C., Saito, Kayoko, Raju, Dheeraj, Benguerba, Kamal, Dabbous, Omar, Mumneh, Nayla, Reyna, Sandra P., Faulkner, Eric

“…We describe outcomes following onasemnogene abeparvovec monotherapy for patients with ≥four survival motor neuron 2 (SMN2) gene copies in RESTORE, a…”
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Withdrawal, attachment security, and recovery from conflict in couple relationships by Prager, Karen J., Poucher, Jesse, Shirvani, Forouz K., Parsons, Julie A., Allam, Zoheb

“…This study used 115 cohabiting couple partners’ 21-day diaries, with which they reported each evening on their moods and their relationships, to test…”
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Electrical impedance myography in duchenne muscular dystrophy and healthy controls: A multicenter study of reliability and validity by Zaidman, Craig M., Wang, Lucy L., Connolly, Anne M., Florence, Julaine, Wong, Brenda L., Parsons, Julie A., Apkon, Susan, Goyal, Namita, Williams, Eugene, Escolar, Diana, Rutkove, Seward B., Bohorquez, Jose L.

“…ABSTRACT Introduction: Electrical impedance myography (EIM) is a non‐invasive, painless, objective technique to quantify muscle pathology. Methods: We measured…”
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Access to novel therapies for Duchenne muscular dystrophy—Insights from expert treating physicians by Aravindhan Veerapandiyan, Anne M. Connolly, Katherine D. Mathews, Stanley Nelson, Craig McDonald, Richard S. Finkel, Vettaikorumakankav Vedanarayanan, Cuixia Tian, Susan Apkon, Julie A. Parsons, Jonathan H. Soslow, William Bryan Burnette, Kaitlin Y. Batley, Susan T. Iannaccone, Carolina Tesi Rocha, Kevin M. Flanigan, Diana Bharucha‐Goebel, Sarah Wright, Migvis Monduy, Simona Treidler, Ashutosh Kumar, Nancy L. Kuntz, Vamshi K. Rao, Rachel Schrader, Saunder M. Bernes, Vikki Ann Stefans, Jena M. Krueger, Marcia V. Felker, Omer Abdul Hamid, Arpita Lakhotia, Susan Matesanz, Partha S. Ghosh, Natalie Katz, Hoda Abdel‐Hamid, Chamindra G. Laverty, Bo Hoon Lee, Amy Harper, Leigh Ramos‐Platt, Diana Castro, Russell J. Butterfield, Crystal M. Proud, Craig M. Zaidman, Emma Ciafaloni

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Consider muscle disease in children with elevated transaminase by Wright, Melissa A, Yang, Michele L, Parsons, Julie A, Westfall, John M, Yee, Audrey S

“…The transaminases alanine aminotransferase (ALT) and aspartate aminotransferase (AST) are markers of hepatocellular injury but are highly concentrated in…”
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West Nile Virus Meningoencephalitis in an Immunocompetent Adolescent by DeBiasi, Roberta L., Parsons, Julie A., Grabert, Brian E.

“…This report describes a case of West Nile virus meningoencephalitis in a previously healthy adolescent. Clinical features included fever, altered mental…”
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Essential Fatty Acid Deficiency Related to the “Amino Acid Diet” in Patients with Spinal Muscular Atrophy (P3.136) by Parsons, Julie A., Watne, Laura

“…Abstract only…”
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Vulnerable Narcissism and Romantic Conflict by Parsons, Julie A

“…Individuals high in vulnerable narcissism often encounter difficulties in their interpersonal relationships. (Lannin et al., 2014; Zeigler-Hill & Besser,…”
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Response: Re: Consider Muscle Disease in Children with Elevated Transaminase by Yee, A. S., Wright, M. A., Yang, M. L., Parsons, J. A., Westfall, J. M.

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