Results 111 to 120 of about 4,206 (188)

Development and Validation of an Ion-Pair Reverse-Phase High-Performance Liquid Chromatography-Electrospray Ionization Mass Spectrometry Method for Determination of Purity of Nusinersen for Quality Control of Drug Substance or Drug Product. [PDF]

Int J Mol Sci
Samoilov M, Zubareva E, Degterev M.
europepmc   +1 more source

Administration practice and adherence of nusinersen in adults with 5q-spinal muscular atrophy in China: an ambispective multicenter study. [PDF]

BMC Neurol
Yao X, Dai Y, Zhu W, Zhao Y, Shang H, Meng Q, Liu Y, Yu L, Hong D, Chen J, Shen L, Chen W, Chen K, Zhang L, Mai M, Cui L.   +15 more
europepmc   +1 more source

Motor function and safety of nusinersen for spinal muscular atrophy: a systematic review and meta-analysis among Chinese patients. [PDF]

Front Pediatr
He G, Feng Z, Li N, Wei S, Liu Y, Tang X.   +5 more
europepmc   +1 more source

MyomiR Networks in Spinal Muscular Atrophy: Associations With Clinical Severity and Treatment Response. [PDF]

Mol Neurobiol
Barbo M, Koritnik B, Leonardis L, Dolžan V, Ravnik-Glavač M.   +4 more
europepmc   +1 more source

Treatment of risdiplam after nusinersen continuously improves upper limb motor function in spinal muscular atrophy patients: a multicenter experience. [PDF]

Front Pediatr
Cheng X, Ma Y, Yu LQ, Fan YB, Zhu LH, Lu HB, Niu Q.   +6 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

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

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

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