Results 321 to 330 of about 120,291 (395)

Systemic mRNA-LNP administration in fetuses improves survival in a mouse model of spinal muscular atrophy. [PDF]

Mol Ther Methods Clin Dev
Carpenter MD, Kus N, Choi RB, Said H, Dumitru AM, Bauer JH, Wang S, Luks V, Teerdhala S, Shah A, Zoltick PW, Alameh MG, Peranteau W.   +12 more
europepmc   +1 more source

Analysis of spinal muscular atrophy patients from the spinal muscular atrophy and muscular dystrophy registry of Pakistan

Bisma Aziz, Ahmed A. Arif, Kulsum Kazi, Salman Kirmani, Zeeshan Ansar, Asghar Nasir, Shahnaz Ibrahim, Karima Y. Ahmed, Zahra Hasan, Sara Khan   +9 more
openalex   +1 more source

Loss of SMN Impairs Osteoblast-Osteoclast Coupling via IGF1-Akt-OPG Axis in Spinal Muscular Atrophy. [PDF]

FASEB J
Xiang T, Zhou Z, Li Y, Suo Y, Dai J, Shi X, Zhou X, Sheng L.   +7 more
europepmc   +1 more source

Respiratory function in 192 adult patients with spinal muscular atrophy (SMA) treated with nusinersen - a multicenter observational study. [PDF]

Orphanet J Rare Dis
Wurster CD, Stolte B, Kessler T, Freigang M, Bjelica B, Ilse B, Koch JC, Cordts I, Mensch A, Zeller D, Uzelac Z, Sam G, Lapp HS, Wohnrade C, Rödiger A, Muhandes MT, Schneider I, Bellut J, Nentwich J, Dorst J, Schuster J, Schreiber-Katz O, Osmanovic A, Totzeck A, Thimm A, Steinbach R, Grosskreutz J, Kleinschnitz C, Ludolph AC, Deschauer M, Kirschner J, Dreyhaupt J, Wollinsky K, Petri S, Weiler M, Hagenacker T, Günther R.   +36 more
europepmc   +1 more source

Neurodevelopmental and mental disorders in children with type I and presymptomatic spinal muscular atrophy. [PDF]

Sci Rep
Buchignani B, Coratti G, Cutrì C, Scattoni ML, Pane M, Palermo C, Leone D, De Sanctis R, Stanca G, Antonaci L, Cutrona C, Pera MC, Brogna C, Cristofani P, Fulceri F, Baranello G, Mercuri E.   +16 more
europepmc   +1 more source

Risdiplam in Spinal Muscular Atrophy: Safety Profile and Use Through The Early Access to Medicine Scheme for the Paediatric Cohort in Great Britain

Nikki Cornell, Anne‐Marie Childs, Elizabeth Wraige, Pinki Munot, Gautam Ambegaonkar, Gabriel Chow, Imelda Hughes, Marjorie Illingworth, Anirban Majumdar, Chiara Marini‐Bettolo, Deepak Parasuraman, Stefan Spinty, Tracey Willis, Mariacristina Scoto, Giovanni Baranello   +14 more
openalex   +2 more sources

Machine learning predicts treatment response to nusinersen in non-sitter Spinal Muscular Atrophy (SMA).

Stimpson G, O'Reilly E, Coratti G, Ridout D, Chakraborti T, Mitra R, de Sanctis R, Pane M, Scoto M, Mercuri E, Muntoni F, Baranello G, International SMA Consortium.   +12 more
europepmc   +1 more source