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

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

Source: In Molecular Genetics and Metabolism Reports December 2024 41

Congenital muscular dystrophies: New aspects of an expanding group of disorders

Authors: Lisi, Matthew T., Cohn, Ronald D.

Source: In BBA - Molecular Basis of Disease 2007 1772(2):159-172

75 - Syndromes, Genetics, and Heritable Heart Disease

Authors: Landis, Benjamin J., Lisi, Matthew T.

Source: In Critical Heart Disease in Infants and Children Edition: Third Edition. 2019:892-904

Contributors

Authors: 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.

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

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

Authors: 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, Judge, Daniel P., Ward, Christopher W., Dietz, Harry C.

Source: Nature Medicine; Feb2007, Vol. 13 Issue 2, p204-210, 7p, 4 Diagrams

Subject Terms: MARFAN syndrome, MUSCLE diseases, ANGIOTENSIN II, TRANSFORMING growth factors-beta, MUSCLE regeneration, MUSCULAR dystrophy

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

Authors: 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

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

Subject Terms: ANGIOTENSINS