Results 111 to 120 of about 1,998 (153)

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. [PDF]

J Neuromuscul Dis
Weiß C, Vill K, Baumann M, Bernert G, Blaschek A, Eisenkölbl A, Flotats-Bastardas M, Friese J, 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, Schara-Schmidt U, Schreiber G, Schwartz O, Sproß J, Stettner GM, Stoltenburg C, Stumpe E, Trollmann R, Wiegand G, Wilichowski E, Kirschner J, Ziegler A, collaborators and members of the INTEGRATE ATMP consortium.   +39 more
europepmc   +1 more source

Open-label phase IV trial evaluating nusinersen after onasemnogene abeparvovec in children with spinal muscular atrophy. [PDF]

J Clin Invest
Proud CM, Finkel RS, Parsons JA, Masson R, Brandsema JF, Kuntz NL, Foster R, Li W, Littauer R, Sohn J, Fradette S, Youn B, Paradis AD, RESPOND Study Group.   +13 more
europepmc   +1 more source

Respiratory outcomes of onasemnogene abeparvovec treatment for spinal muscular atrophy: national real-world cohort study. [PDF]

Eur J Pediatr
Lavie M, Rochman M, Armoni Domany K, Golan Tripto I, Be'er M, Besor O, Sagi L, Aharoni S, Ginsberg M, Noyman I, Levine H.   +10 more
europepmc   +1 more source

Onasemnogene Abeparvovec Gene Therapy and Risdiplam for the Treatment of Spinal Muscular Atrophy in Thailand: A Cost-Utility Analysis. [PDF]

Appl Health Econ Health Policy
Khuntha S, Prawjaeng J, Ponragdee K, Sanmaneechai O, Srinonprasert V, Leelahavarong P.   +5 more
europepmc   +1 more source

Treating Presymptomatic Spinal Muscular Atrophy Patients with Onasemnogene Abeparvovec in Italy: The Role of the National Health System and Drug Supply. Comment on Zaidman et al. Newborn Screening for Spinal Muscular Atrophy: Variations in Practice and Early Management of Infants with Spinal Muscular Atrophy in the United States. <i>Int. J. Neonatal Screen.</i> 2024, <i>10</i>, 58. [PDF]

Int J Neonatal Screen
Masson R, Gaballo S, Caravita R, Parravicini S.   +3 more
europepmc   +1 more source

Real-world multidisciplinary outcomes of onasemnogene abeparvovec monotherapy in patients with spinal muscular atrophy type 1: experience of the French cohort in the first three years of treatment. [PDF]

Orphanet J Rare Dis
Desguerre I, Barrois R, Audic F, Barnerias C, Chabrol B, Davion JB, Durigneux J, Espil-Taris C, Gomez-Garcia de la Banda M, Guichard M, Isapof A, Nougues MC, Laugel V, Le Goff L, Mercier S, Pervillé A, Richelme C, Thibaud M, Sarret C, Schweitzer C, Testard H, Trommsdorff V, Vanhulle C, Walther-Louvier U, Altuzarra C, Chouchane M, Ropars J, Quijano-Roy S, Cances C.   +28 more
europepmc   +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