Results 181 to 190 of about 6,859 (228)

Newborn Screening for Spinal Muscular Atrophy in the UK: Use of Modelling to Identify Priorities for Ongoing Evaluation. [PDF]

Int J Neonatal Screen
Thokala P, Bessey A, Knowles R, Marshall J, Visintin C, Lawton M, Lombardo S.   +6 more
europepmc   +1 more source

Application of Biomarkers in Spinal Muscular Atrophy. [PDF]

Int J Mol Sci
Gao C, Zhan Y, Chen H, Deng C.
europepmc   +1 more source

Reduction in Perioperative Risk in Patients with Spinal Muscular Atrophy Following the Release of Disease-Modifying Therapies: An Analysis of the National Surgical Quality Improvement Program Database. [PDF]

Children (Basel)
Toaz E, Pinto N, Kilner K, Cheon E.
europepmc   +1 more source

The efficacy and safety of Nusinersen for spinal muscular atrophy types 1, 2, 3: a systematic review of the current evidence [PDF]

Somaia Daghriri, Yara A Alorfi, Rasil Sulaiman Alayed, Wid F. Altaf, Ghaida E. Alharbi, Hisham Abdulrahman Al-Sulami, Raghad Alkhalifah, Omar Alzahrani, Abdulrahman Softah   +8 more
openalex   +1 more source

Assessment of Fine Motor Abilities Among Children with Spinal Muscular Atrophy Treated with Nusinersen Using a New Touchscreen Application: A Pilot Study. [PDF]

Children (Basel)
Klemm I, Danial-Saad A, Karlin AR, Nassar-Yassien R, Eshel I, Levine H, Steinberg T, Aharoni S.   +7 more
europepmc   +1 more source

Neurofilaments as Biomarkers of the Efficacy of Risdiplam Treatment in Early SMA Phenotypes Diagnosed by Newborn Screening. [PDF]

Children (Basel)
Pitarch-Castellano I, Ñungo-Garzón NC, Aragon-Gawińska K, Ibáñez-Albert E, Vázquez-Costa JF, Sevilla T.   +5 more
europepmc   +1 more source

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

Mehmet Akif Kılıç, Fulya Kürekçi, Osman Kipoğlu, Orhan Coşkun, Burçin Nazlı Karacabey, Selahattin Katar, Rıdvan Avci, Hülya Maraş Genç, Erkan Çakır, Edibe Pembegül Yıldız   +9 more
openalex   +2 more sources

Cerebrospinal fluid metabolomics reveals predictive biomarkers of nusinersen therapy efficacy in type II and type III spinal muscular atrophy patients. [PDF]

Neurol Sci
Li D, Sun N, Wu H, Wang X, Shi Y, Yang L, Huang S, Zhang K, Yang C.   +8 more
europepmc   +1 more source

Evaluating Therapeutic Outcomes in Spinal Muscular Atrophy: An Indian Experience. [PDF]

Ann Indian Acad Neurol
Mohanlal S, Mubeena PM, Hussain H, Anand M, Kumar S, Ashraf VV, Rabia TA, Janardhanan S, Kumar S.   +8 more
europepmc   +1 more source

Difficulties of Eating and Masticating Solid Food in Children with Spinal Muscular Atrophy-Preliminary Study. [PDF]

Nutrients
Winnicka E, Łabuz A, Kułaga Z, Grochowski T, Socha P.   +4 more
europepmc   +1 more source