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The New England Journal of Medicine, ISSN 0028-4793, 11/2017, Volume 377, Issue 18, pp. 1713 - 1722
Journal Article
Journal of neuromuscular diseases, ISSN 2214-3599, 2019, Volume 6, Issue 3, p. 307
Spinal muscular atrophy type 1 (SMA1) is the leading genetic cause of infant mortality for which therapies, including AVXS-101 (onasemnogene abeparvovec,... 
Cell survival | Action potential | Infants | Infant mortality | Gene therapy | Age | Natural history | Spinal muscular atrophy
Journal Article
Journal of Neurology, Neurosurgery & Psychiatry, ISSN 0022-3050, 07/2019, Volume 90, Issue e7, pp. A5 - A6
Journal Article
by Maffioletti, S M and Sarcar, S and Henderson, A and Mannhardt, I and Pinton, L and Moyle, L A and Moyle, L A and Steele-Stallard, H and Cappellari, O and Wells, K E and Ragazzi, M and Ragazzi, M and Ragazzi, M and Wang, W and Wang, W and Zammit, P and Wells, D J and Eschenhagen, T and Jung-Klawitter, S and Fuchs, N V and Upton, K R and Frömmrich, A and Miskey, C and Muñoz-Lopez, M and Shukla, R and Wang, J and Sebe, A and Merkert, S and Bock, A and Held, U and Menzel, M and Gogol-Döring, A and Haase, A and Izsvák, Z and Izsvák, Z and Ivics, Z and Martin, U and Garcia-Perez, J and Faulkner, G J and Terrasso, A P and Silva, M M and Arez, F and Sousa, M F and Raimundo, N and Gomes-Alves, P and Kremer, E J and Alves, P M and Brito, C and Moser, R and Decembrini, S and Gamm, D and Gamm, D and de Leone, V Ponce and Alves, S and Christaller, W and Subashi, E and Kacher, R and Lamazière, A and Despres, G and Saudou, F and Caboche, J and Betuing, S and Miyauchi, A and Nakajima, T and Taga, N and Ono, S A and Mizukami, H and Kato, M and Osaka, H and Muramatsu, S and Yamagata, T and Bouquet, C and Pruneau, D and Pruneau, D and Thomasson, N and Thomasson, N and Chavas, J and Chavas, J and Galluppi, F and Dalkara, D and Besnosman, R and Chenegros, G and Picaud, S and Sahel, J and Massó, A and Sánchez, A and Bosch, A and Blanch, R and Espinosa, J F and Giménez-Llort, L and Heidbüchel, J and Veinalde, R and Veinalde, R and Veinalde, R and Jäger, D and Jäger, D and Jäger, D and von Kalle, C and von Kalle, C and von Kalle, C and ...
Human Gene Therapy, ISSN 1043-0342, 12/2017, Volume 28, Issue 12, pp. A1 - A125
Journal Article
Schizophrenia Research, ISSN 0920-9964, 2008, Volume 106, Issue 2, pp. 300 - 307
Journal Article
01/2018, ISBN 9781351084536
Book Chapter
Journal of the American College of Cardiology, ISSN 0735-1097, 03/2003, Volume 41, Issue 6, pp. 291 - 292
Journal Article
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