Wildon Farwell

225P Initial data from the DELIVER trial of DYNE-251 in males with DMD Mutations amenable to Exon 51 skipping

L. De Waele, Craig Campbell, N. Deconinck, K. Flanigan et autres

221P Initial data from the achieve trial of DYNE-101 in adults with myotonic dystrophy type 1 (DM1)

Douglas C. Wolf, James B Lilleker, Guillaume Bassez, Jordi Díaz‐Manera et autres

P50 A phase 1/2 randomized, placebo-controlled, multiple ascending dose study (ACHIEVE) of DYNE-101 in individuals with myotonic dystrophy type 1 (DM1)

Douglas C. Wolf, Chris Mix, Baoqin Han, A. Dugar et autres

Identifying Biomarkers of Spinal Muscular Atrophy for Further Development

Jacqueline Glascock, Basil T. Darras, Thomas O. Crawford, Charlotte J. Sumner et autres

gb, us (code pays fourni par la source)

Correction to: Comparative All-Cause Mortality Among a Large Population of Patients with Spinal Muscular Atrophy Versus Matched Controls

Emma Viscidi, Maneesh Juneja, Jin Wang, Nasha Wang et autres

us, gb (code pays fourni par la source)

Corrigendum to ‘Population pharmacokinetics-based recommendations for a single delayed or missed dose of nusinersen’

Drew MacCannell, Zdenek Berger, Lilly East, Eugenio Mercuri et autres

us, it, de, gb, au (code pays fourni par la source)

Comparative All-Cause Mortality Among a Large Population of Patients with Spinal Muscular Atrophy Versus Matched Controls

Emma Viscidi, Maneesh Juneja, Jin Wang, Nasha Wang et autres

us, gb (code pays fourni par la source)

Implications of circulating neurofilaments for spinal muscular atrophy treatment early in life: A case series

Christiano R. R. Alves, Marco Petrillo, Rebecca Spellman, Reid Garner et autres

us (code pays fourni par la source)

Nusinersen by subcutaneous intrathecal catheter for symptomatic spinal muscular atrophy patients with complex spine anatomy

Vincent J. Carson, Millie Young, Karlla W. Brigatti, Robert M. Reed et autres

us (code pays fourni par la source)

Treatment of infantile-onset spinal muscular atrophy with nusinersen: final report of a phase 2, open-label, multicentre, dose-escalation study

Richard S. Finkel, Claudia A. Chiriboga, Jiri Vajsar, Jacqueline Montes et autres

us, ca, gb (code pays fourni par la source)

Increased systemic HSP70B levels in spinal muscular atrophy infants

Eric J. Eichelberger, Christiano R. R. Alves, Ren Zhang, Marco Petrillo et autres

us (code pays fourni par la source)

Plasma Phosphorylated Neurofilament Heavy Chain (pNF-H) Level is Associated with Future Motor Function in Nusinersen-treated Individuals with Later-onset Spinal Muscular Atrophy (SMA) (2248)

Michelle A. Farrar, Francesco Muntoni, Charlotte J. Sumner, Thomas O. Crawford et autres

au, gb, us (code pays fourni par la source)