M176 SUBCUTANEOUS C1-INHIBITOR FACILITATES CONTINUITY OF HEREDITARY ANGIOEDEMA MANAGEMENT FROM CHILDHOOD TO ADOLESCENCE von Mumneh, N., Chapovsky, F.

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P02 Real-world data for patients with gestational age ≤35 weeks at birth treated with onasemnogene abeparvovec: results from the RESTORE Registry von Finkel, R., Dabbous, O., Benguerba, K., Mumneh, N., Raju, D., Servais, L.

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P231 Early intervention and speed-to-effect in spinal muscular atrophy type 1 following onasemnogene abeparvovec gene replacement therapy von Toro, W., Reyna, S., Ritter, S., Patel, A., Mumneh, N., Dabbous, O.

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P79 Real-world outcomes of disease-modifying treatment for patients with spinal muscular atrophy: findings from a global retrospective chart review von Dabbous, O., LaMarca, N., Toro, W., Wallach, S., Mumneh, N., Aassi, M., O'Brien, E., Baranello, G., Reyna, S.

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161P Long-term safety of onasemnogene abeparvovec for patients with spinal muscular atrophy: real-world findings from the RESTORE Registry von Servais, L., Alecu, I., Lopez-Leon, S., Ilic, A., Brueckner, A., Benguerba, K., Reyna, S., Dabbous, O., Mumneh, N., Finkel, R.

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258P Safety and tolerability of onasemnogene abeparvovec for patients with spinal muscular atrophy weighing ≤17 kg and ≤24 months old: phase 4 OFELIA study von Zanoteli, E., Muntadas, J., Gurgel-Gianetti, J., Monges, S., Aliberti, P., Alecu, I., Ritter, S., de Lana, J. Martins, Mumneh, N., Saute, J.

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M178 SUBCUTANEOUS ADMINISTRATION FACILITATES INITIATION OF C1-INHIBITOR PROPHYLAXIS IN PATIENTS WITH HEREDITARY ANGIOEDEMA AND FREQUENT ATTACKS von Li, H., Mumneh, N., Chiao, J.

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Fixed-Dose Subcutaneous C1-Inhibitor Liquid for Prophylactic Treatment of C1-INH-HAE: SAHARA Randomized Study von Lumry, William R., Martinez-Saguer, Inmaculada, Yang, William H., Bernstein, Jonathan A., Jacobs, Joshua, Moldovan, Dumitru, Riedl, Marc A., Johnston, Douglas T., Li, H. Henry, Tang, Yongqiang, Schranz, Jennifer, Lu, Peng, Vardi, Moshe, Farkas, Henriette, Keith, P., Yang, W., Maurer, M., Martinez-Saguer, I., Farkas, H., Reshef, A., Kivity, S., Moldovan, D., Caballero, T., Guilarte, M., Hernandez, M.D., González-Quevedo, M.T., Banerji, A., Bernstein, J., Bewtra, A., Craig, T., Fineman, S., Gower, R., Jacobs, J., Johnston, D., Kashkin, J., Li, H.H., Lumry, W.R., Manning, M., McNeil, D., Melamed, I., Mumneh, N., Nickel, T., Panuto, J., Soteres, D., Tachdjian, R., Offenberger, J., Wedner, J.

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Restoration of immunity with interleukin-2 therapy von Smith, K A, Jacobson, E L, Emert, R, Giordano, M, Kovacs, E, Mumneh, N, Pilaro, F, Sohn, T, Warren, D

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