Results 161 to 170 of about 3,107 (205)

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

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

Emerging thrombotic disorders associated with virus-based innovative therapies: from VITT to AAV-gene therapy-related thrombotic microangiopathy [PDF]

Benemei, Silvia, Marcucci, Rossella
core   +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

Onasemnogene abeparvovec for spinal muscular atrophy.

Australian prescriber
openaire   +1 more source

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Onasemnogene-abeparvovec

Reactions Weekly
Stephanie J. Phelps, Tracy M. Hagemann, Kelley R. Lee   +2 more
semanticscholar   +12 more sources

Onasemnogene abeparvovec

Reactions Weekly, 2023
  +7 more sources

The efficacy of gene therapy with onasemnogene abeparvovec in spinal muscular atrophy in young patients

L O Badalyan Neurological Journal
Introduction. The advent of pathogenetic therapy has significantly changed the prognosis for spinal muscular atrophy (SMA). However, our knowledge of the pathogenetic methods of treating SMA is mostly based on the results of clinical trials, which are ...
D. A. Fisenko, A. L. Kurenkov, L. Kuzenkova, V. Chernikov, E. V. Uvakina, Sofya G. Popovich, B. Bursagova, L. M. Abdullaeva, Julia A. Kurova, Nadezhda S. Adalimova, Dar`ya S. Nikolenko, O. V. Globa, Natalya V. Andreenko   +12 more
semanticscholar   +1 more source