Results 141 to 150 of about 4,206 (188)

Intrathecal onasemnogene abeparvovec for treatment-experienced patients with spinal muscular atrophy: a phase 3b, open-label trial. [PDF]

Nat Med
Kwon JM, Munell F, Le Goff L, Yuge K, Kato T, Cances C, De Waele L, Woodcock IR, Mercuri EM, Proud CM, Darras BT, Hayes LH, Oskoui M, Visootsak J, Williams G, Ilić A, Yang L, van der Pol WL.   +17 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

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

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

Nusinersen for spinal muscular atrophy.

Australian prescriber
openaire   +1 more source

Nusinersen for adults with spinal muscular atrophy

Neurological Sciences, 2023
Nusinersen was effective in improving motor function and survival in infantile and childhood-onset spinal muscular atrophy (SMA), and the value of real-world experiences in adult SMA patients increase gradually. Here, we present our clinical experience in adult SMA patients treated with nusinersen according to CHERISH study.Thirty-two SMA patients ...
Doruk Arslan, Berin Inan, Muhammed Kilinc   +2 more
exaly   +4 more sources

Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy [PDF]

New England Journal of Medicine, 2018
BACKGROUND: Nusinersen is an antisense oligonucleotide drug that modulates pre-messenger RNA splicing of the survival motor neuron 2 (SMN2) gene. It has been developed for the treatment of spinal muscular atrophy (SMA).
Eugenio Mercuri, Basil T Darras, Claudia A Chiriboga   +2 more
exaly   +3 more sources