Five-Year Extension Results of the Phase 1 START Trial of Onasemnogene Abeparvovec in Spinal Muscular Atrophy von Mendell, Jerry R, Al-Zaidy, Samiah A, Lehman, Kelly J, McColly, Markus, Lowes, Linda P, Alfano, Lindsay N, Reash, Natalie F, Iammarino, Megan A, Church, Kathleen R, Kleyn, Aaron, Meriggioli, Matthew N, Shell, Richard

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AVXS-101 (Onasemnogene Abeparvovec) for SMA1: Comparative Study with a Prospective Natural History Cohort von Al-Zaidy, Samiah A., Kolb, Stephen J., Lowes, Linda, Alfano, Lindsay N., Shell, Richard, Church, Kathleen R., Nagendran, Sukumar, Sproule, Douglas M., Feltner, Douglas E., Wells, Courtney, Ogrinc, Francis, Menier, Melissa, L’Italien, James, Arnold, W. David, Kissel, John T., Kaspar, Brian K., Mendell, Jerry R.

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Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy von Mendell, Jerry R, Al-Zaidy, Samiah, Shell, Richard, Arnold, W. Dave, Rodino-Klapac, Louise R, Prior, Thomas W, Lowes, Linda, Alfano, Lindsay, Berry, Katherine, Church, Kathleen, Kissel, John T, Nagendran, Sukumar, L’Italien, James, Sproule, Douglas M, Wells, Courtney, Cardenas, Jessica A, Heitzer, Marjet D, Kaspar, Allan, Corcoran, Sarah, Braun, Lyndsey, Likhite, Shibi, Miranda, Carlos, Meyer, Kathrin, Foust, K.D, Burghes, Arthur H.M, Kaspar, Brian K

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Follistatin Gene Therapy for Sporadic Inclusion Body Myositis Improves Functional Outcomes von Mendell, Jerry R., Sahenk, Zarife, Al-Zaidy, Samiah, Rodino-Klapac, Louise R., Lowes, Linda P., Alfano, Lindsay N., Berry, Katherine, Miller, Natalie, Yalvac, Mehmet, Dvorchik, Igor, Moore-Clingenpeel, Melissa, Flanigan, Kevin M., Church, Kathleen, Shontz, Kim, Curry, Choumpree, Lewis, Sarah, McColly, Markus, Hogan, Mark J., Kaspar, Brian K.

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Assessment of Systemic Delivery of rAAVrh74.MHCK7.micro-dystrophin in Children With Duchenne Muscular Dystrophy: A Nonrandomized Controlled Trial von Mendell, Jerry R, Sahenk, Zarife, Lehman, Kelly, Nease, Carrie, Lowes, Linda P, Miller, Natalie F, Iammarino, Megan A, Alfano, Lindsay N, Nicholl, Amanda, Al-Zaidy, Samiah, Lewis, Sarah, Church, Kathleen, Shell, Richard, Cripe, Linda H, Potter, Rachael A, Griffin, Danielle A, Pozsgai, Eric, Dugar, Ashish, Hogan, Mark, Rodino-Klapac, Louise R

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Whole-genome sequencing of patients with rare diseases in a national health system von Turro, Ernest, Astle, William J., Megy, Karyn, Gräf, Stefan, Greene, Daniel, Shamardina, Olga, Allen, Hana Lango, Sanchis-Juan, Alba, Frontini, Mattia, Thys, Chantal, Stephens, Jonathan, Mapeta, Rutendo, Burren, Oliver S., Downes, Kate, Haimel, Matthias, Tuna, Salih, Deevi, Sri V. V., Aitman, Timothy J., Bennett, David L., Calleja, Paul, Carss, Keren, Caulfield, Mark J., Chinnery, Patrick F., Dixon, Peter H., Gale, Daniel P., James, Roger, Koziell, Ania, Laffan, Michael A., Levine, Adam P., Maher, Eamonn R., Markus, Hugh S., Morales, Joannella, Morrell, Nicholas W., Mumford, Andrew D., Ormondroyd, Elizabeth, Rankin, Stuart, Rendon, Augusto, Richardson, Sylvia, Roberts, Irene, Roy, Noemi B. A., Saleem, Moin A., Smith, Kenneth G. C., Stark, Hannah, Tan, Rhea Y. Y., Themistocleous, Andreas C., Thrasher, Adrian J., Watkins, Hugh, Webster, Andrew R., Wilkins, Martin R., Williamson, Catherine, Whitworth, James, Humphray, Sean, Bentley, David R., Kingston, Nathalie, Walker, Neil, Bradley, John R., Ashford, Sofie, Penkett, Christopher J., Freson, Kathleen, Stirrups, Kathleen E., Raymond, F. Lucy, Ouwehand, Willem H.

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Australian sea levels—Trends, regional variability and influencing factors von White, Neil J., Haigh, Ivan D., Church, John A., Koen, Terry, Watson, Christopher S., Pritchard, Tim R., Watson, Phil J., Burgette, Reed J., McInnes, Kathleen L., You, Zai-Jin, Zhang, Xuebin, Tregoning, Paul

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Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy von Mendell, Jerry R., Al-Zaidy, Samiah, Shell, Richard, Arnold, W. Dave, Rodino-Klapac, Louise R., Prior, Thomas W., Lowes, Linda, Alfano, Lindsay, Berry, Katherine, Church, Kathleen, Kissel, John T., Nagendran, Sukumar, L’Italien, James, Sproule, Douglas M., Wells, Courtney, Cardenas, Jessica A., Heitzer, Marjet D., Kaspar, Allan, Corcoran, Sarah, Braun, Lyndsey, Likhite, Shibi, Miranda, Carlos, Meyer, Kathrin, Foust, K.D., Burghes, Arthur H.M., Kaspar, Brian K.

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A comprehensive library of human transcription factors for cell fate engineering von Ng, Alex H. M., Khoshakhlagh, Parastoo, Rojo Arias, Jesus Eduardo, Pasquini, Giovanni, Wang, Kai, Swiersy, Anka, Shipman, Seth L., Appleton, Evan, Kiaee, Kiavash, Kohman, Richie E., Vernet, Andyna, Dysart, Matthew, Leeper, Kathleen, Saylor, Wren, Huang, Jeremy Y., Graveline, Amanda, Taipale, Jussi, Hill, David E., Vidal, Marc, Melero-Martin, Juan M., Busskamp, Volker, Church, George M.

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Berlin Heart EXCOR and ACTION post-approval surveillance study report von Zafar, Farhan, Conway, Jennifer, Bleiweis, Mark S., Al-Aklabi, Mohammed, Ameduri, Rebecca, Barnes, Aliessa, Bearl, David W., Buchholz, Holger, Church, Stephanie, Do, Nhue L., Duffy, Vicky, Dykes, John C., Eghtesady, Pirooz, Fisher, Lauren, Friedland-Little, Joshua, Fuller, Stephanie, Fynn-Thompson, Francis, George, Kristen, Gossett, Jeffrey G., Griffiths, Eric R., Griselli, Massimo, Hawkins, Beth, Honjo, Osami, Jeewa, Aamir, Joong, Anna, Kindel, Steven, Kouretas, Peter, Lorts, Angela, Machado, Desiree, Maeda, Katsuhide, Maurich, Andrea, May, Lindsay J., McConnell, Patrick, Mehegan, Mary, Mongé, Michael, Morales, David L.S., Murray, Jenna, Niebler, Robert A., O'Connor, Matthew, Peng, David M., Phelps, Christina, Philip, Joseph, Ploutz, Michelle, Profsky, Michael, Reichhold, Allison, Rosenthal, David N., Said, Ahmed S., Schumacher, Kurt R., Si, Ming-Sing, Simpson, Kathleen E., Sparks, Joshua, Louis, Jim St, Steiner, Marie E., VanderPluym, Christina, Villa, Chet

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Long‐term safety and functional outcomes of delandistrogene moxeparvovec gene therapy in patients with Duchenne muscular dystrophy: A phase 1/2a nonrandomized trial von Mendell, Jerry R., Sahenk, Zarife, Lehman, Kelly J., Lowes, Linda P., Reash, Natalie F., Iammarino, Megan A., Alfano, Lindsay N., Lewis, Sarah, Church, Kathleen, Shell, Richard, Potter, Rachael A., Griffin, Danielle A., Hogan, Mark, Wang, Shufang, Mason, Stefanie, Darton, Eddie, Rodino‐Klapac, Louise R.

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Whole-genome sequencing of a sporadic primary immunodeficiency cohort von Thaventhiran, James E. D., Lango Allen, Hana, Burren, Oliver S., Rae, William, Greene, Daniel, Staples, Emily, Zhang, Zinan, Farmery, James H. R., Simeoni, Ilenia, Rivers, Elizabeth, Maimaris, Jesmeen, Penkett, Christopher J., Stephens, Jonathan, Deevi, Sri V. V., Sanchis-Juan, Alba, Gleadall, Nicholas S., Thomas, Moira J., Sargur, Ravishankar B., Gordins, Pavels, Baxendale, Helen E., Brown, Matthew, Tuijnenburg, Paul, Worth, Austen, Hanson, Steven, Linger, Rachel J., Buckland, Matthew S., Rayner-Matthews, Paula J., Gilmour, Kimberly C., Samarghitean, Crina, Seneviratne, Suranjith L., Sansom, David M., Lynch, Andy G., Megy, Karyn, Ellinghaus, Eva, Ellinghaus, David, Jorgensen, Silje F., Karlsen, Tom H., Stirrups, Kathleen E., Cutler, Antony J., Kumararatne, Dinakantha S., Chandra, Anita, Edgar, J. David M., Herwadkar, Archana, Cooper, Nichola, Grigoriadou, Sofia, Huissoon, Aarnoud P., Goddard, Sarah, Jolles, Stephen, Schuetz, Catharina, Boschann, Felix, Lyons, Paul A., Hurles, Matthew E., Savic, Sinisa, Burns, Siobhan O., Kuijpers, Taco W., Turro, Ernest, Ouwehand, Willem H., Thrasher, Adrian J., Smith, Kenneth G. C.

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Health outcomes in spinal muscular atrophy type 1 following AVXS‐101 gene replacement therapy von Al‐Zaidy, Samiah, Pickard, A. Simon, Kotha, Kavitha, Alfano, Lindsay N., Lowes, Linda, Paul, Grace, Church, Kathleen, Lehman, Kelly, Sproule, Douglas M., Dabbous, Omar, Maru, Benit, Berry, Katherine, Arnold, W. David, Kissel, John T., Mendell, Jerry R., Shell, Richard

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Germline selection shapes human mitochondrial DNA diversity von Wei, Wei, Tuna, Salih, Keogh, Michael J, Smith, Katherine R, Aitman, Timothy J, Beales, Phil L, Bennett, David L, Gale, Daniel P, Bitner-Glindzicz, Maria A K, Black, Graeme C, Brennan, Paul, Elliott, Perry, Flinter, Frances A, Floto, R Andres, Houlden, Henry, Irving, Melita, Koziell, Ania, Maher, Eamonn R, Markus, Hugh S, Morrell, Nicholas W, Newman, William G, Roberts, Irene, Sayer, John A, Smith, Kenneth G C, Taylor, Jenny C, Watkins, Hugh, Webster, Andrew R, Wilkie, Andrew O M, Williamson, Catherine, Ashford, Sofie, Penkett, Christopher J, Stirrups, Kathleen E, Rendon, Augusto, Ouwehand, Willem H, Bradley, John R, Raymond, F Lucy, Caulfield, Mark, Turro, Ernest, Chinnery, Patrick F

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Comprehensive Rare Variant Analysis via Whole-Genome Sequencing to Determine the Molecular Pathology of Inherited Retinal Disease von Stephens, Jonathan, Dewhurst, Eleanor, Malka, Samantha, Plagnol, Vincent, Rizzo, Roberta, Scott, Richard H., Henderson, Robert H.H., MacLaren, Robert E., Paterson, Joan, Aitman, Timothy, Ali, Sonia, Ambegaonkar, Gautum, Arno, Gavin, Astle, William, Attwood, Antony, Bennett, David, Bitner-Glindzicz, Maria, Bleda, Marta, Boggard, Harm, Carss, Keren, Clements-Brod, Naomi, DaCosta, Rosa, De Vries, Minka, Dewhurst, Eleanor, Drewe, Elizabeth, Egner, William, Erber, Wendy N., Everington, Tamara, Fletcher, Debra, Freson, Kathleen, Gale, Daniel, Ghali, Neeti, Ghurye, Rohit, Gräf, Stefan, Greene, Daniel, Grigoriadou, Sofia, Grozeva, Detelina, Hackett, Scott, Hadinnapola, Charaka, Hague, Rosie, Hammerton, Tracey, Heemskerk, Johan W.M., Holder, Muriel, Holder, Susan, Huissoon, Aarnoud, Hurst, Jane, Jolles, Stephen, Keeling, David, Kennedy, Fiona, Kiely, David, Lawrie, Allan, Lear, Sara, Lees, Melissa, Lentaigne, Claire, Lorenzo, Lorena, Mangles, Sarah, Mapeta, Rutendo, Masati, Larahmie, Mathias, Mary, Michaelides, Michel, Millar, Carolyn M., Moledina, Shahin, Moore, Anthony, Murng, Sai, Oksenhendler, Eric, Park, Soo-Mi, Patch, Chris, Paterson, Joan, Penkett, Christopher J., Pepke-Zaba, Joanna, Pollock, Val, Qasim, Waseem, Quinti, Isabella, Reid, Evan, Rondina, Matthew, Rosser, Elisabeth, Santra, Saikat, Sargur, Ravishankar, Savic, Sinisa, Scully, Marie, Sewell, Carrock, Smith, Kenneth, Southgate, Laura, Stauss, Hans, Stein, Penelope, Talks, Kate, Thomas, Ellen, Thrasher, Adrian, Tischkowitz, Marc, Titterton, Catherine, Turro, Ernest, von Ziegenweldt, Julie, Vonk Noordegraaf, Anton, Wakeling, Emma, Wanjiku, Ivy, Welch, Steve, Westbury, Sarah, Woods, Geoffrey, Yong, Patrick, Webster, Andrew R.

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Gene therapy with bidridistrogene xeboparvovec for limb-girdle muscular dystrophy type 2E/R4: phase 1/2 trial results von Mendell, Jerry R., Pozsgai, Eric R., Lewis, Sarah, Griffin, Danielle A., Lowes, Linda P., Alfano, Lindsay N., Lehman, Kelly J., Church, Kathleen, Reash, Natalie F., Iammarino, Megan A., Sabo, Brenna, Potter, Rachael, Neuhaus, Sarah, Li, Xiaoxi, Stevenson, Herb, Rodino-Klapac, Louise R.

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Positive Fluid Balance Is Associated with Higher Mortality and Prolonged Mechanical Ventilation in Pediatric Patients with Acute Lung Injury von Flori, Heidi R., Church, Gwynne, Liu, Kathleen D., Gildengorin, Ginny, Matthay, Michael A.

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Phenotypic Characterization of EIF2AK4 Mutation Carriers in a Large Cohort of Patients Diagnosed Clinically With Pulmonary Arterial Hypertension von Hadinnapola, Charaka, Bleda, Marta, Haimel, Matthias, Screaton, Nicholas, Swift, Andrew, Dorfmüller, Peter, Preston, Stephen D, Southwood, Mark, Hernandez-Sanchez, Jules, Martin, Jennifer, Treacy, Carmen, Yates, Katherine, Bogaard, Harm, Church, Colin, Coghlan, Gerry, Condliffe, Robin, Corris, Paul A, Gibbs, Simon, Girerd, Barbara, Holden, Simon, Humbert, Marc, Kiely, David G, Lawrie, Allan, Machado, Rajiv, MacKenzie Ross, Robert, Moledina, Shahin, Montani, David, Newnham, Michael, Peacock, Andrew, Pepke-Zaba, Joanna, Rayner-Matthews, Paula, Shamardina, Olga, Soubrier, Florent, Southgate, Laura, Suntharalingam, Jay, Toshner, Mark, Trembath, Richard, Vonk Noordegraaf, Anton, Wilkins, Martin R, Wort, Stephen J, Wharton, John, Gräf, Stefan, Morrell, Nicholas W

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Gene Delivery for Limb-Girdle Muscular Dystrophy Type 2D by Isolated Limb Infusion von Mendell, Jerry R, Chicoine, Louis G, Al-Zaidy, Samiah A, Sahenk, Zarife, Lehman, Kelly, Lowes, Linda, Miller, Natalie, Alfano, Lindsay, Galliers, Beverly, Lewis, Sarah, Murrey, Darren, Peterson, Ellyn, Griffin, Danielle A, Church, Kathleen, Cheatham, Sharon, Cheatham, John, Hogan, Mark J, Rodino-Klapac, Louise R

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Genome sequence of Xenia2 a DV cluster phage that infects Gordonia rubripertincta von Agaiby, Carol, Ahmed, Maha, Argueta, Aidan, Arrowood, Kyle, Barrier, Keelynn P, Church, Meghan W, Connell, Cheryl R, Dao, Ken D, Dao, Kathleen Huyen T, Davenport, Makenzie R, Edmondson, Megan D, Estabrook, Makenzie I, Gondhi, Santoshi, Gonzalez, Patricia, Leduc, Francine, Ma, Trang, Mansoor, Adam, Mansoor, Sara, Mattley, Lillian, Meyer, Cyrus, Nguyen, Loc, Niaz, Emaan, Parker, Jenna M, Ross, Delaney C, Scott, Devin M, Semryck, Brianna, Takla, Kyrillos, Tiramdas, Aishwarya, Upputuru, Sai Kaushik, Pollenz, Richard S

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