Results 101 to 110 of about 1,998 (153)

Intrathecal onasemnogene abeparvovec for treatment-experienced patients with spinal muscular atrophy: a phase 3b, open-label trial. [PDF]

Nat Med
Kwon JM, Munell F, Le Goff L, Yuge K, Kato T, Cances C, De Waele L, Woodcock IR, Mercuri EM, Proud CM, Darras BT, Hayes LH, Oskoui M, Visootsak J, Williams G, Ilić A, Yang L, van der Pol WL.   +17 more
europepmc   +1 more source

Administration of Onasemnogene Abeparvovec in an Infant With Spinal Muscular Atrophy and PCR-Confirmed SARS-CoV-2 Infection. [PDF]

Cureus
Shimomura I, Maruyama S, Kodama Y, Matsunaga M, Okamoto Y.   +4 more
europepmc   +1 more source

Trach and treat: Safety and motor outcomes following onasemnogene abeparvovec in patients with spinal muscular atrophy and tracheostomies in the RESTORE registry. [PDF]

J Neuromuscul Dis
Erbaş Y, Servais L, Shieh PB, Goedeker NL, Waldrop MA, Bo R, Raju D, Benguerba K, Reyna SP, Wolff D, Finkel RS.   +10 more
europepmc   +1 more source

Changes in electrophysiological findings of spinal muscular atrophy type I after the administration of nusinersen and onasemnogene abeparvovec: two case reports. [PDF]

BMC Neurol, 2023
Mizuno T, Kanouchi T, Tamura Y, Hirata K, Emoto R, Suzuki T, Kashimada K, Morio T.   +7 more
europepmc   +1 more source

Intrathecal onasemnogene abeparvovec in treatment-naive patients with spinal muscular atrophy: a phase 3, randomized controlled trial. [PDF]

Nat Med
Proud CM, Vũ DC, Wilmshurst JM, Sanmaneechai O, Gulati S, Xiong H, Moreno HC, Tay SKH, Thong MK, Born AP, Banzzatto Ortega A, Jong YJ, Al-Muhaizea MA, Lee AW, Visootsak J, Tauscher-Wisniewski S, Alecu I, Parlikar R, Finkel RS, STEER Study Group.   +19 more
europepmc   +1 more source

Early Development of Spinal Deformities in Children Severely Affected with Spinal Muscular Atrophy after Gene Therapy with Onasemnogene Abeparvovec-Preliminary Results. [PDF]

Children (Basel), 2023
Soini V, Schreiber G, Wilken B, Hell AK.
europepmc   +1 more source

Safety and efficacy of intravenous onasemnogene abeparvovec gene therapy in patients with spinal muscular atrophy type 1: interim analysis from LT-001, a long-term follow-up study of patients from the START study. [PDF]

EClinicalMedicine
Waldrop MA, Escudero RB, Yang L, Camarata RA, Branic EC, Mehl L, Ilić A, Connolly AM.   +7 more
europepmc   +1 more source

Risdiplam treatment following onasemnogene abeparvovec in individuals with spinal muscular atrophy: a multicenter case series. [PDF]

BMC Neurol
Svoboda MD, Kuntz N, Leon-Astudillo C, Byrne BJ, Krueger J, Kwon JM, Sieburg C, Castro D.   +7 more
europepmc   +1 more source

Reduction of Preexisting AAV9 Antibody Titers Before Onasemnogene Abeparvovec Administration in Twins With Spinal Muscular Atrophy. [PDF]

Neurology
Nelson RW, Cox CA, Stark M, Johnson AD, Sichmeller K, Earhart J, Ihinger JM, Stafki SA, Karachunski P, Kang PB.   +9 more
europepmc   +1 more source

Fatal outcomes following onasemnogene abeparvovec in advanced-stage spinal muscular atrophy. [PDF]

Gene Ther
Pongsakornkullachart P, Kulsirichawaroj P, Kongkasuwan R, Tovichien P, Jitwongwai S, Kanjanauthai S, Preeprem N, Limpaninlachat S, Sermpon N, Sanmaneechai O.   +9 more
europepmc   +1 more source