Results 161 to 170 of about 1,722 (231)

Sensory dysfunction in SMA type 2 and 3 - adaptive mechanism or concomitant target of damage? [PDF]

Orphanet J Rare Dis
Koszewicz M, Ubysz J, Dziadkowiak E, Wieczorek M, Budrewicz S.   +4 more
europepmc   +1 more source

MOLECULAR ANALYSIS OF CHILDHOOD-ONSET SPINAL MUSCULAR-ATROPHY

, 2016
Theodosiou, A, Rodrigues, N, Talbot, K, Campbell, L, Nesbit, M, Owen, N, Ambrose, H, Muntoni, F, Patel, S, Ignatius, J, Dubowitz, V, Davies, K   +11 more
openaire   +1 more source

Longitudinal data collection in pediatric and adult patients with 5q spinal muscular atrophy in Latin America: LATAM RegistrAME study - a clinical registry study protocol. [PDF]

Einstein (Sao Paulo)
Batista EC, Zanoteli E, Monfardini F, Santos GPD, Silva GS, Berwanger O, Rizzo LV, Fonseca HARD.   +7 more
europepmc   +1 more source

Bulbar function in children with spinal muscular atrophy type 1 treated with nusinersen. [PDF]

Dev Med Child Neurol
Stimpson G, Fanelli L, Conway E, Johnson E, Berti B, Scoto M, Muntoni F, Mercuri E, Baranello G, p‐FOIS/CEDAS and OrSAT Working Group.   +9 more
europepmc   +1 more source

Ubiquitination Insight from Spinal Muscular Atrophy-From Pathogenesis to Therapy: A Muscle Perspective. [PDF]

Int J Mol Sci
Bolado-Carrancio A, Tapia O, Rodríguez-Rey JC.   +2 more
europepmc   +1 more source

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

Safety and Efficacy of Nusinersen Focusing on Renal and Hematological Parameters in Spinal Muscular Atrophy. [PDF]

Brain Behav
Bahadır Şenol H, Yıldız G, Polat Aİ, Aydın A, Hız AS, Soylu A, Yiş U.   +6 more
europepmc   +1 more source

Cost comparison analysis of onasemnogene abeparvovec and nusinersen for treatment of patients with spinal muscular atrophy type 1 in the Netherlands. [PDF]

Eur J Health Econ
van der Schans S, Velikanova R, Weidlich D, Howells R, Patel A, Bischof M, Postma MJ, Boersma C.   +7 more
europepmc   +1 more source

Towards a Definition of Physiologic Vulnerability in Pediatric Spine Surgery: Identification of Key Risk Factors in a Cohort Study of Children With Neuromuscular Disease Undergoing Spinal Fusion. [PDF]

Global Spine J
Ghauri MS, Rajkumar S, Stone LE, Kelly MP, Iyer RR, Bauer J, Ames CP, Newton PO, Gonda DD, Levy ML, Ravindra VM.   +10 more
europepmc   +1 more source

Open-label phase IV trial evaluating nusinersen after onasemnogene abeparvovec in children with spinal muscular atrophy. [PDF]

J Clin Invest
Proud CM, Finkel RS, Parsons JA, Masson R, Brandsema JF, Kuntz NL, Foster R, Li W, Littauer R, Sohn J, Fradette S, Youn B, Paradis AD, RESPOND Study Group.   +13 more
europepmc   +1 more source