Results 161 to 170 of about 2,700 (197)

EE424 Societal Costs of Spinal Muscular Atrophy Type 1 for Patients Treated with Onasemnogene Abeparvovec or Nusinersen in the United Kingdom [PDF]

J. Kleintjens, Anita Patel, Marjolijn van Keep, K. K. Srivastava, Philip McHale, Silvia Affinito, M Bischof   +6 more
openalex   +1 more source

Real-World Safety Data of the Orphan Drug Onasemnogene Abeparvovec (Zolgensma^®) for the SMA Rare Disease: A Pharmacovigilance Study Based on the EMA Adverse Event Reporting System. [PDF]

Pharmaceuticals (Basel)
Ruggiero R, Balzano N, Nicoletti MM, di Mauro G, Fraenza F, Campitiello MR, Rossi F, Capuano A.   +7 more
europepmc   +1 more source

Onasemnogene Abeparvovec: Post-infusion Efficacy and Safety in Patients With Spinal Muscular Atrophy (SMA)-A Fondazione Policlinico Gemelli IRCCS Experience. [PDF]

Hosp Pharm
Favia M, Tarantino D, Cerbo LD, Sabia A, Campopiano R, Pani M.   +5 more
europepmc   +1 more source

Table 2_Real-world outcomes of spinal muscular atrophy treatment with onasemnogene abeparvovec in Croatia: a comprehensive case series and literature review.docx

Ivan Lehman (21590618), Matija Matošević (14619110), Lovro Lamot (674823), Branka Bunoza (21590621)   +3 more
openalex   +1 more source

Delphi consensus on gene therapy of spinal muscular atrophy with onasemnogene abeparvovec in Germany, Austria and Switzerland-part I-systematic literature review and existing evidence

Claudia Weiß, Katharina Vill, Matthias Baumann, Günther Bernert, Astrid Blaschek, Eisenkölbl, Astrid, Marina Flotats Bastardas, Johannes Friese, Claudia Ganter, Klaus Goldhahn, Andreas Hahn, von der Hagen, Maja, Hans Hartmann, Oswald Hasselmann, Veronka Horber, Ralf A Husain, Sabine Illsinger, David Jacquier, Jessika Johannsen, Cornelia Köhler, Heike Kölbel, Michael Kolodzig, Andrea Klein, Astrid Pechmann, Arpad von Moers, Wolfgang Müller-Felber, Christian Rauscher, Ulrike Schara-Schmidt, Gudrun Schreiber, Stettner, Georg M, al, et,   +30 more
openalex   +1 more source

Onasemnogene abeparvovec for spinal muscular atrophy.

Australian prescriber
openaire   +1 more source

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Onasemnogene Abeparvovec-xioi: Gene Therapy for Spinal Muscular Atrophy

Annals of Pharmacotherapy, 2020
Objective: To review the efficacy and safety of onasemnogene abeparvovec-xioi (Zolgensma) in the treatment of spinal muscular atrophy (SMA). Data Sources: An English-language literature search of PubMed, MEDLINE, and Ovid (1946 to December 2019) was completed using the terms onasemnogene, AVXS-101, and spinal muscular atrophy.
Debra Stevens, Melanie K. Claborn, Brooke L. Gildon, Tiffany L. Kessler, Cheri Walker   +4 more
openaire   +4 more sources

Onasemnogene-abeparvovec

Reactions Weekly
Stephanie J. Phelps, Tracy M. Hagemann, Kelley R. Lee   +2 more
  +9 more sources