Results 111 to 120 of about 3,596 (160)

The role of vascular pathology in SMA: implications for pathogenesis and treatment. [PDF]

Front Cell Neurosci
Parson SH, Allardyce H.
europepmc   +1 more source

The effectiveness and value of therapies for spinal muscular atrophy. [PDF]

J Manag Care Spec Pharm
Tice JA, Luu L, Sanchez S, Lee W, Nikitin D, Phillips M, Carlson JJ, Chan HH, Rind DM, Agboola F.   +9 more
europepmc   +1 more source

Matched-pair analysis of motor outcomes in adults with spinal muscular atrophy on nusinersen vs. risdiplam. [PDF]

J Neurol
Neuhoff S, Stolte B, Lipka J, Schlag M, Pul R, Schmitt LI, Leo M, Skuljec J, Deuschl C, Forsting M, Kleinschnitz C, Hagenacker T.   +11 more
europepmc   +1 more source

Rare SMA Patients: A Comprehensive Look at Clinical Features, Genetic Profiles and Therapeutic Approaches. [PDF]

Int J Mol Sci
Mikhalchuk K, Artemieva S, Zabnenkova V, Akhkiamova M, Dadali E, Rudenskaya G, Sparber P, Rybakova O, Papina Y, Monakhova A, Shulyakova I, Saiko D, Zhiteneva S, Polyakov A, Shchagina O.   +14 more
europepmc   +1 more source

Enhanced splicing modulation by NMA-modified antisense oligonucleotides. [PDF]

Nucleic Acids Res
Ling K, Prakash TP, Yu J, Jackson M, Chun SJ, Bachmann G, Post N, Greenlee S, Soriano A, Hunyara JL, Norris DA, Jafar-Nejad P, Swayze EE, Bennett CF, Rigo F.   +14 more
europepmc   +1 more source

Real-world treatment patterns and unmet needs in spinal muscular atrophy: a caregiver-centric survey study from China. [PDF]

BMC Neurol
Fan W, Zheng WE, Li J, Wang R, Ni S, Xue P, Xu C.   +6 more
europepmc   +1 more source

High-dose nusinersen for spinal muscular atrophy: a phase 3 randomized trial. [PDF]

Nat Med
Finkel RS, Crawford TO, Mercuri E, Sumner CJ, Garcia Romero MDM, Day JW, Montes J, Sun P, Tichler B, Paradis AD, Boesch E, Inra J, Littauer R, Sohn J, Monine M, Gambino G, Foster R, Farewell R, Fradette S.   +18 more
europepmc   +1 more source

Electrophysiological Changes in Pediatric Spinal Muscular Atrophy: Results From an Observational Study. [PDF]

Muscle Nerve
Sun R, Wang J, Han J, Jiang J, Wu J, Chen X, Deng X, Cai C, Sun D.   +8 more
europepmc   +1 more source

Nusinersen rescues taurine deficiency in patients with type 1 Spinal Muscular Atrophy. [PDF]

Commun Med (Lond)
di Vito R, Hassan A, Nuzzo T, Caretto A, Panicucci C, Bruno C, Bertini E, D'Amico A, Vercelli A, Boido M, Errico F, Pellizzoni L, Usiello A.   +12 more
europepmc   +1 more source