Results 181 to 190 of about 1,822 (274)

The long-term efficacy of nusinersen on respiratory functions in children with symptomatic spinal muscular atrophy type 1. [PDF]

open access: yesTurk J Med Sci
Kiliç MA   +9 more
europepmc   +1 more source

Diagnostic Journey of Korean Patients with Spinal Muscular Atrophy. [PDF]

open access: yesYonsei Med J
Kim SH   +6 more
europepmc   +1 more source

Brazilian Portuguese version of the revised upper-limb module: cross-cultural adaptation and validation. [PDF]

open access: yesArq Neuropsiquiatr
Artilheiro MC   +8 more
europepmc   +1 more source

Clinical and Genetic Profiles of 5q- and Non-5q-Spinal Muscular Atrophy Diseases in Pediatric Patients. [PDF]

open access: yesGenes (Basel)
Nishio H   +7 more
europepmc   +1 more source

Respiratory outcomes of onasemnogene abeparvovec treatment for spinal muscular atrophy: national real-world cohort study. [PDF]

open access: yesEur J Pediatr
Lavie M   +10 more
europepmc   +1 more source

Treatment acceptability for disease‐modifying therapy for type 1 diabetes (T1D)—Views from parents of children with presymptomatic T1D

open access: yes
Diabetes, Obesity and Metabolism, Volume 27, Issue 10, Page 6059-6067, October 2025.
Lauren M. Quinn   +8 more
wiley   +1 more source

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