Combination therapy with nusinersen and AVXS-101 in SMA type 1

Affiliations

¹ From Pediatric Neurology (B.H.L.), Department of Neurology (E.C., L.L., D.G., K.E., E.C.), and Department of Pediatric Pulmonology (K.V.), University of Rochester, NY; and Pediatric Neurology (H.Z.A.-H.), Children's Hospital of Pittsburgh of UPMC, PA.
² From Pediatric Neurology (B.H.L.), Department of Neurology (E.C., L.L., D.G., K.E., E.C.), and Department of Pediatric Pulmonology (K.V.), University of Rochester, NY; and Pediatric Neurology (H.Z.A.-H.), Children's Hospital of Pittsburgh of UPMC, PA. emma_ciafaloni@urmc.rochester.edu.

Case Reports

Bo Hoon Lee et al. Neurology. 2019.

. 2019 Oct 1;93(14):640-641.

doi: 10.1212/WNL.0000000000008207. Epub 2019 Sep 5.

¹ From Pediatric Neurology (B.H.L.), Department of Neurology (E.C., L.L., D.G., K.E., E.C.), and Department of Pediatric Pulmonology (K.V.), University of Rochester, NY; and Pediatric Neurology (H.Z.A.-H.), Children's Hospital of Pittsburgh of UPMC, PA.
² From Pediatric Neurology (B.H.L.), Department of Neurology (E.C., L.L., D.G., K.E., E.C.), and Department of Pediatric Pulmonology (K.V.), University of Rochester, NY; and Pediatric Neurology (H.Z.A.-H.), Children's Hospital of Pittsburgh of UPMC, PA. emma_ciafaloni@urmc.rochester.edu.

No abstract available

Comment in

Spinal muscular atrophy: Further expanding the clinician's armamentarium.
Brandsema JF, Sumner CJ. Brandsema JF, et al. Neurology. 2019 Oct 1;93(14):613-614. doi: 10.1212/WNL.0000000000008192. Epub 2019 Sep 5. Neurology. 2019. PMID: 31488614 No abstract available.