Gene therapy for spinal muscular atrophy: the Qatari experience von Ali, Hossamaldein Gaber, Ibrahim, Khalid, Elsaid, Mahmoud Fawzi, Mohamed, Reem Babiker, Abeidah, Mahmoud I. A., Al Rawwas, Azhar Othman, Elshafey, Khaled, Almulla, Hajer, El-Akouri, Karen, Almulla, Mariam, Othman, Amna, Musa, Sara, Al-Mesaifri, Fatma, Ali, Rehab, Shahbeck, Noora, Al-Mureikhi, Mariam, Alsulaiman, Reem, Alkaabi, Saad, Ben-Omran, Tawfeg

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Mutation in noncoding RNA RNU12 causes early onset cerebellar ataxia von Elsaid, Mahmoud Fawzi, Chalhoub, Nader, Ben‐Omran, Tawfeg, Kumar, Pankaj, Kamel, Hussein, Ibrahim, Khalid, Mohamoud, Yasmin, Al‐Dous, Eman, Al‐Azwani, Iman, Malek, Joel A., Suhre, Karsten, Ross, M. Elizabeth, Aleem, Alice Abdel

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Altered PLP 1 splicing causes hypomyelination of early myelinating structures von Kevelam, Sietske H, Taube, Jennifer R, Rosalina M. L. van Spaendonk, Bertini, Enrico, Sperle, Karen, Tarnopolsky, Mark, Tonduti, Davide, Valente, Enza Maria, Travaglini, Lorena, Sistermans, Erik A, Bernard, Geneviève, Coriene E. Catsman‐Berrevoets, Clara D. M. van Karnebeek, Østergaard, John R, Friederich, Richard L, Mahmoud Fawzi Elsaid, Schieving, Jolanda H, Maja Tarailo‐Graovac, Orcesi, Simona, Steenweg, Marjan E, Carola G. M. van Berkel, Waisfisz, Quinten, Abbink, Truus E M, Marjo S. van der Knaap, Hobson, Grace M, Wolf, Nicole I

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