Optimizing clinical outcomes: The journey of twins with CRIM-negative infantile-onset Pompe disease on high-dose enzyme replacement therapy and immunomodulation

Autor: Fares, Angie H., Desai, Ankit K., Case, Laura E., Sharon, Cassie, Klinepeter, Amy, Kirby, Amelia, Lisi, Matthew T., Koch, Rebecca L., Kishnani, Priya S.

Publikováno v: In Molecular Genetics and Metabolism Reports December 2024 41

Contributors

Autor: Ahmed, Mubbasheer, Alaish, Samuel M., Alanmanou, Euleche, Alexander, Plato, Aljiffry, Alaa, Almodovar, Melvin C., Alsoufi, Bahaaldin, Anders, Marc M., Andersen, Nicholas D., Ascenzi, Judith, Aydin, Scott I., Bacon, Matthew K., Barron, David J., Basken, Amy, Beddows, Kimberly D., Bembea, Melania M., Benscoter, Alexis L., Bergstrom, Charles P., Bernier, Meghan, Bibevski, Steve, Bichell, David, Bird, Geoffrey L., Boukas, Konstantinos, Bove, Edward L., Brady, Ken, Broberg, Craig S., Bronicki, Ronald A., Brothers, Julie A., Brown, Kristen M., Brownlee, John R., Bryant, Roosevelt, III, Buddhavarapu, Amulya, Cameron, Duke E., Chai, Paul J., Checchia, Paul A., Cheifetz, Ira M., Chin, Clifford, Cholette, Jill Marie, Cole, Charles R., Cooper, David S., Coulson, John D., Damiano, Ralph J., Jr., DeLeon, Miguel, DeSena, Holly C., Deutsch, Nina, Eghtesady, Pirooz, Engorn, Branden, Everett, Allen, Felmly, Lloyd, Fiore, Andrew C., Fleming, Gregory A., Flores, Saul, Franklin, Rodney, Fraser, Charles D., III, Gaies, Michael, Gangemi, James J., Gaur, Lasya, Ghanayem, Nancy S., Ginde, Salil, Gist, Katja M., Goldman, Allan, Goldstein, Stuart L., Goswami, Dheeraj, Graham, Eric M., Grenier, Michelle A., Handler, Stephanie S., Herlong, James R., Hill, Kevin D., Romano, Jennifer C., Ho, Siew Yen, Hoffman, George M., Honjo, Osami, Hornik, Christoph P., Hsu, Daphne T., Huddleston, Charles B., Huff, Christin, Hunt, Elizabeth A., Idriss, Salim F., Iliopoulos, Ilias, Jackson, Kimberly Ward, Jacobs, Jeffrey P., Jacobs, Marshall L., Jaggers, James, Jansen, Laura N., Janson, Christopher M., Jaquiss, Robert, Johnson, Emily, Jones, Melissa B., Justice, Lindsey, Kane, Patricia L., Karamlou, Tara, Kartha, Vyas M., Kavarana, Minoo N., Khan, Abigail May, King, Valerie, Kirsch, Roxanne E., Kirshbom, Paul M., Knott-Craig, Christopher J., Koo, Jeannie, Kramer, Jennifer, Krawczeski, Catherine D., Krishnamurthy, Ganga, Kudchadkar, Sapna R., Kumar, Karan R., Kumar, T.K. Susheel, Kwiatkowski, David M., Lamour, Jacqueline M., Lancaster, Timothy S., Landis, Benjamin J., Lasa, Javier J., Liava'a, Matthew H.L., Licht, Daniel J., Lisi, Matthew T., Loftin, Ryan, Loomba, Rohit S., Marino, Bradley S., Maxey, Thomas S., McCarthy, Karen, McCrory, Michael C., Mehta, Inder D., Mehta, Christopher, Meliones, Jon N., Meliones, Christine, Miles, Alison, Mitchell, Michael E., Molitor-Kirsch, Erica, Montgomery, Jenny A., Moore, Lisa, Morales, David L.S., Morin, Cara, Morin, Nicholas, Mou, Steven S., Muralidaran, Ashok, Murthy, Raghav, Nellis, Joseph R., Nelson, Jennifer S., Nelson McMillan, Kristen, Nies, Melanie, Nigro, John, Noje, Corina, Norris, Sarah E., O'Brien, James, Ofori-Amanfo, George, Ohye, Richard G., Ootaki, Yoshio, Ozment, Caroline P., Peek, Giles J., Peterson, Autumn K., Peterson, Renuka E., Petty, John K., Porayette, Prashob, Procaccini, David E., Quintessenza, James, Ragalie, William S., Ravekes, William, Raymond, Tia T., Redington, Andrew, Rehder, Kyle J., Riggs, Becky, Rivera, Ramon Julio, Roark, Jennifer, Romer, Lewis H., Ryan, Amy, Ryan, Thomas D., Rymeski, Beth A., Sassalos, Peter, Sawyer, Jaclyn E., Scholl, Frank, Schooler, Kevin Patrick, Schuette, Jennifer, McElrath, Jamie, Sedehi, Daniel R., Sekar, Priya, Shaffner, Donald H., Shah, Sanket, Shen, Irving, Shetty, Avinash K., Shope, Edd, Shores, Darla, Si, Ming-Sing, Siddiqi, Nida, Simpson, Leah, Slavik, Zdenek, Smith, Heidi A.B., Spector, Zebulon Z., Speer, Allison L., Spevak, Philip, Stewart, Dylan, Stewart, Robert D., St. Louis, James, Stone, Matthew L., Su, Erik, Swain, Kelly A., Takemoto, Cliff M., Tallent, Sarah, Thiagarajan, Ravi R., Traube, Chani, Tropp, Ephraim, Tsang, Rocky, Tume, Sebastian C., Turek, Joseph W., Turi, Jennifer L., Turner, Immanuel I., Tweddell, James S., Unegbu, Chinwe, Ungerleider, Ross M., Ungerleider, Jamie Dickey, Ungerleider, Graham D., Vricella, Luca A., Vu, Eric L., Wadia, Rajeev S., Walsh, Michael J., Watt, Kevin M., Welke, Karl, Willett, Renée, Williams, Derek A., Woods, Ronald K., Woods-Hill, Charlotte, Yarrabolu, Tharakanatha R.

Publikováno v: In Critical Heart Disease in Infants and Children Edition: Third Edition. 2019:vi-xvii

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Corrigendum: Angiotensin II type 1 receptor blockade attenuates TGF-β–induced failure of muscle regeneration in multiple myopathic states.

Autor: Cohn, Ronald D., van Erp, Christel, Habashi, Jennifer P., Soleimani, Arshia A., Klein, Erin C., Lisi, Matthew T., Gamradt, Matthew, ap Rhys, Colette M., Holm, Tammy M., Loeys, Bart L., Ramirez, Francesco., J.udge, Daniel P, Ward, Christopher W, Dietz, Harry C

Publikováno v: Nature Medicine; Apr2007, Vol. 13 Issue 4, p511-511, 1p