Results 101 to 110 of about 3,114 (145)

Comparative Clinical Outcomes of Nusinersen and Gene Therapy in Spinal Muscular Atrophy Type 1.

JAMA Netw Open
Ropars J, Cances C, Garcia-Uzquiano R, Gomez-Garcia de la Banda M, Barnerias C, Audic F, Durigneux J, Halbert C, Nkam L, Laugel V, Espil C, Walther-Louvier U, Davion JB, Isapof A, Le Goff L, Desguerre I, Quijano-Roy S, Grimaldi L, French SMA Registry Study Group.   +18 more
europepmc   +1 more source

Disease characteristics and treatment status of genetically confirmed spinal muscular atrophy patients: a cross-sectional survey in China. [PDF]

BMC Neurol
Zhang D, Yao F, Ling C, Jiang S, Yang X, Dai Y.   +5 more
europepmc   +1 more source

The 2025 Oligo Meeting in Budapest: Highlights of the 21^st annual meeting of the oligonucleotide therapeutics society. [PDF]

Mol Ther Nucleic Acids
Godinho BMDC, Guo S.
europepmc   +1 more source

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

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

Effectiveness of Risdiplam Treatment in Adult Patients With Spinal Muscular Atrophy Type IIb-III. [PDF]

Rev Neurol
García Estévez DA.
europepmc   +1 more source