Search Results - "Proud, Crystal"

Expert recommendations and clinical considerations in the use of onasemnogene abeparvovec gene therapy for spinal muscular atrophy by Kichula, Elizabeth A., Proud, Crystal M., Farrar, Michelle A., Kwon, Jennifer M., Saito, Kayoko, Desguerre, Isabelle, McMillan, Hugh J.

“…Spinal muscular atrophy (SMA) is an autosomal recessive, neurodegenerative disease caused by biallelic mutations in the survival motor neuron 1 (SMN1) gene…”
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50 Years Ago in The Journal of Pediatrics: Pseudohypertrophic Muscular Dystrophy: Duchenne Type by Proud, Crystal M

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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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Delandistrogene Moxeparvovec Gene Therapy in Ambulatory Patients (Aged ≥4 to <8 Years) with Duchenne Muscular Dystrophy: 1‐Year Interim Results from Study SRP‐9001‐103 (ENDEAVOR) by Zaidman, Craig M., Proud, Crystal M., McDonald, Craig M., Lehman, Kelly J., Goedeker, Natalie L., Mason, Stefanie, Murphy, Alexander P., Guridi, Maitea, Wang, Shufang, Reid, Carol, Darton, Eddie, Wandel, Christoph, Lewis, Sarah, Malhotra, Jyoti, Griffin, Danielle A., Potter, Rachael A., Rodino‐Klapac, Louise R., Mendell, Jerry R.

“…Objective Delandistrogene moxeparvovec is approved in the USA for the treatment of ambulatory patients (4–5 years) with Duchenne muscular dystrophy. ENDEAVOR…”
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Equitable Access of Delandistrogene Moxeparvovec for Patients With Duchenne Muscular Dystrophy: A Call for Discussion by Hamid, Omer Abdul, Hester, D. Micah, Matesanz, Susan E., Wright, Sarah, Batley, Kaitlin Y., Proud, Crystal M., Veerapandiyan, Aravindhan

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50 years ago in the Journal of Pediatrics: the use of serum creatine phosphokinase and other serum enzymes in the diagnosis of progressive muscular dystrophy by Proud, Crystal M

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Examining Real-World Adherence to Nusinersen for the Treatment of Spinal Muscular Atrophy Using Two Large US Data Sources by Youn, Bora, Proud, Crystal M., Wang, Nasha, Hou, Qiang, Viscidi, Emma, Eaton, Susan, Paradis, Angela D., Neville, Bridget A., Johnson, Nicole B.

“…Introduction Spinal muscular atrophy (SMA) is a rare neuromuscular disease characterized by progressive muscular atrophy and weakness. Nusinersen was the first…”
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50 years ago in the Journal of Pediatrics: Photogenic and self-induced epilepsy by Proud, Crystal M

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Combination disease‐modifying treatment in spinal muscular atrophy: A proposed classification by Proud, Crystal M., Mercuri, Eugenio, Finkel, Richard S., Kirschner, Janbernd, De Vivo, Darryl C., Muntoni, Francesco, Saito, Kayoko, Tizzano, Eduardo F., Desguerre, Isabelle, Quijano‐Roy, Susana, Benguerba, Kamal, Raju, Dheeraj, Faulkner, Eric, Servais, Laurent

“…Abstract We sought to devise a rational, systematic approach for defining/grouping survival motor neuron ‐targeted disease‐modifying treatment (DMT) scenarios…”
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Spinal Muscular Atrophy Update in Best Practices: Recommendations for Treatment Considerations by Schroth, Mary K, Deans, Jennifer, Bharucha Goebel, Diana X, Burnette, W Bryan, Darras, Basil T, Elsheikh, Bakri H, Felker, Marcia V, Klein, Andrea, Krueger, Jena, Proud, Crystal M, Veerapandiyan, Aravindhan, Graham, Robert J

“…Spinal muscular atrophy (SMA) is an autosomal recessive disorder caused by biallelic variants of the gene ( ) that affects approximately 1 in 15,000 live…”
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Management of Select Adverse Events Following Delandistrogene Moxeparvovec Gene Therapy for Patients With Duchenne Muscular Dystrophy by Zaidman, Craig M, Goedeker, Natalie L, Aqul, Amal A, Butterfield, Russell J, Connolly, Anne M, Crystal, Ronald G, Godwin, Kara E, Hor, Kan N, Mathews, Katherine D, Proud, Crystal M, Kula Smyth, Elizabeth, Veerapandiyan, Aravindhan, Watkins, Paul B, Mendell, Jerry R

“…Duchenne muscular dystrophy (DMD) is a rare, degenerative, recessive X-linked neuromuscular disease. Mutations in the gene encoding dystrophin lead to the…”
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O19: Integrated analyses of data from clinical trials of delandistrogene moxeparvovec gene therapy in DMD by O’Rourke, Erin, Proud, Crystal, Zaidman, Craig, Shieh, Perry B., McDonald, Craig, Day, John W., Mason, Stefanie, Guridi, Maitea, Han, Lixin, Yu, Lixi, Reid, Carol, Darton, Eddie, Wandel, Christoph, Richardson, James, Malhotra, Jyoti, Singh, Teji, Rodino-Klapac, Louise, Mendell, Jerry

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50 Years Ago in TheJournal ofPediatrics by Proud, Crystal M.

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50 Years Ago in T J P by Proud, Crystal M.

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50 Years Ago in The Journal of Pediatrics by Proud, Crystal M.

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50 Years Ago in The Journal of Pediatrics by Proud, Crystal M.

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Baseline Characteristics and Interim Safety in RESPOND: A Phase 4 Study in Children with Spinal Muscular Atrophy (SMA) Treated With Nusinersen After Onasemnogene Abeparvovec (P7-9.003) by Kuntz, Nancy, Parsons, Julie, Brandsema, John F., Proud, Crystal, Finkel, Richard, Swoboda, Kathryn, Masson, Ricardo, Liu, Yingying, Makepeace, Corinne, Paradis, Angela, Berger, Zdenek, Somera-Molina, Kathleen

“…Abstract only…”
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Spinraza in Adults with Spinal Muscular Atrophy (SAS) – 14 month results (S33.010) by Zaidman, Craig, Proud, Crystal, Thonhoff, Jason, Rad, Nassim, Ho, Doreen, Chu, MaryLynn, Ladha, Shafeeq, Crawford, Thomas, Nayar, Shakti, Genge, Angela, Frey, Margaret, Heatwole, Chad, Lew, Daphne

“…Abstract only…”
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Baseline Characteristics and Initial Safety Results in RESPOND: A Phase 4 Study of Nusinersen in Children with Spinal Muscular Atrophy (SMA) Who Received Onasemnogene Abeparvovec (P18-5.003) by Brandsema, John, Parsons, Julie, Proud, Crystal, Finkel, Richard, Swoboda, Kathryn, Masson, Ricardo, Liu, Yingying, Makepeace, Corinne, Paradis, Angela, Berger, Zdenek, Wagner, Joanne, Somera-Molina, Kathleen

“…Abstract only…”
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Practical Considerations for Delandistrogene Moxeparvovec Gene Therapy in Patients With Duchenne Muscular Dystrophy by Mendell, Jerry R., Proud, Crystal, Zaidman, Craig M., Mason, Stefanie, Darton, Eddie, Wang, Shufang, Wandel, Christoph, Murphy, Alexander P., Mercuri, Eugenio, Muntoni, Francesco, McDonald, Craig M.

“…Delandistrogene moxeparvovec is a gene transfer therapy approved in the United States, United Arab Emirates, and Qatar for the treatment of ambulatory patients…”
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