Results 111 to 120 of about 1,977 (181)

Therapeutic strategies for spinal muscular atrophy: the history and future perspective. [PDF]

Front Hum Neurosci
Liu J, Liu Y, Yuan A, Ning K.
europepmc   +1 more source

Delphi consensus on gene therapy of spinal muscular atrophy with onasemnogene abeparvovec in Germany, Austria and Switzerland - part II - expert based recommendations for surveillance and management of side-effects. [PDF]

J Neuromuscul Dis
Ziegler A, Weiß C, Vill K, Baumann M, Bernert G, Blaschek A, Eisenkölbl A, Flotats-Bastardas M, Friese J, Holzwarth D, Ganter C, Goldhahn K, Hahn A, von der Hagen M, Hartmann H, Hasselmann O, Horber V, Husain RA, Illsinger S, Jacquier D, Johannsen J, Köhler C, Kölbel H, Kolodzig M, Klein A, Pechmann A, von Moers A, Müller-Felber W, Rauscher C, Schreiber G, Schwartz O, Sproß J, Stettner GM, Stoltenburg C, Stumpe E, Trollmann R, Wiegand G, Wilichowski E, Schara U, Kirschner J, collaborators and members of the INTEGRATE ATMP consortium.   +40 more
europepmc   +1 more source

Risdiplam therapy in adult patients with Spinal muscular Atrophy (SMA). A 24-month-real-world experience at a single muscular centre. [PDF]

Acta Myol
Cesarone E, Picillo E, Scutifero M, Passamano L, Nigro V.   +4 more
europepmc   +1 more source

Specificity and exon target space of splicing modifying compounds. [PDF]

Nat Commun
Lenkeit F, Knehr J, Altorfer M, Byrnes A, Li W, Hsiao J, Wu C, Gaitonde P, Skaanderup PR, Mullin S, Solovyeva E, Pikusa M, Krueger AT, Ottl J, Keller CG, Kolter C, Naumann U, Ottis P, Reyes A.   +18 more
europepmc   +1 more source

Long-term persistence, safety and effectiveness of nusinersen in spinal muscular atrophy: a population-based study. [PDF]

J Neurol
Aragon-Gawinska K, Nungo-Garzon NC, Muelas N, Sivera R, Sevilla T, Hervas D, Pitarch-Castellano I, Vazquez-Costa JF.   +7 more
europepmc   +1 more source

In Utero Molecular-Targeted Drug Therapies: Translational Principles, Pharmacologic Considerations, and Emerging Clinical Applications. [PDF]

J Clin Med
Hasegawa A, Rojhani E, Fathallah AH, Ruano R, Shamshirsaz AA.   +4 more
europepmc   +1 more source

Longitudinal motor function and biomarker correlates in treated adult spinal muscular atrophy: a single-center cohort study. [PDF]

Front Neurol
Yano C, Ando M, Yoshimura A, Higuchi Y, Noda Y, Yuan JH, Nakamura T, Hobara T, Nagatomo R, Kojima F, Yuji M, Hiramatsu Y, Nozuma S, Hashiguchi A, Sakiyama Y, Takashima H.   +15 more
europepmc   +1 more source

Evaluating effects of risdiplam in adults with spinal muscular atrophy: a monocentric study. [PDF]

ERJ Open Res
Crescimanno G, Alonge P, Lupica A, Tomasello V, Di Stefano V, Brighina F, Marrone O.   +6 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

Treatment evolution in spinal muscular atrophy: insights from the SMArtCARE registry. [PDF]

Brain
Voigt-Müller C, Pfaffenlehner M, Bernert G, Cetin H, Hagenacker T, Kölbel H, Lochmüller H, Pfeuffer C, Vill K, Walter MC, Kirschner J, Pechmann A, SMArtCARE study group .   +12 more
europepmc   +1 more source